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Raquitismo hipofosfatêmico
ORPHA:437CID-10 · E83.31CID-11 · 5C63.22DOENÇA RARA
Esta doença tem causa genética. Recomendamos procurar um médico geneticista para diagnóstico, exames genéticos e aconselhamento familiar.
Sistema ósseoInício todas idadesHerança AD/AR/XL/XLD
Sinônimos clínicos: Raquitismo resistente à vitamina D

O raquitismo hipofosfatêmico é um grupo de doenças genéticas caracterizadas por hipofosfatemia, raquitismo e níveis séricos normais de cálcio.

Última verificação: Fonte: não verificado (1 pendência)Em construçãohistóricoSugerir correção
Mantido por Agente Raras·Colaborar como especialista →

Introdução

O que você precisa saber de cara

💬
EM LINGUAGEM SIMPLES

A hipofosfatemia ligada ao X é caracterizada por baixos níveis de fosfato no sangue (hipofosfatemia), o que pode levar a anomalias esqueléticas. O fosfato é um mineral essencial para o desenvolvimento normal dos ossos e dos dentes. Sem quantidades adequadas de fosfato, os ossos podem enfraquecer ou amolecer, levando a uma condição chamada raquitismo em crianças em crescimento ou a uma condição semelhante chamada osteomalácia em adultos.

As características da hipofosfatemia ligada ao X variam amplamente, mesmo entre membros da mesma família. Indivíduos levemente afetados podem ter hipofosfatemia sem outros sinais e sintomas. Indivíduos mais gravemente afetados tipicamente apresentam crescimento lento a partir da primeira infância, e podem ser mais baixos que seus pares. Eles frequentemente desenvolvem anomalias ósseas que podem prejudicar o movimento e causar dor, como pernas anormalmente curvadas (arqueadas) porque os ossos são fracos demais para suportar peso. Essas anomalias tendem a piorar com o tempo.

Em adultos com hipofosfatemia ligada ao X, a osteomalácia pode tornar os ossos mais propensos a um tipo de fratura chamada fratura por estresse, que pode ser dolorosa. Adultos afetados também podem apresentar depósitos de cálcio anormais que ocorrem próximo às articulações onde ligamentos e tendões se inserem (entesiopatia) e um distúrbio articular doloroso chamado osteoartrite.

Outros sinais e sintomas da hipofosfatemia ligada ao X podem incluir fusão prematura dos ossos do crânio (craniossinostose) e perda auditiva. Indivíduos afetados também podem ter anomalias dentárias, que podem incluir pus causado por uma infecção bacteriana (abscesso). Em casos raros, pessoas com hipofosfatemia ligada ao X apresentam anomalias da coluna vertebral, que podem incluir estreitamento do canal vertebral que pode comprimir a parte superior da medula espinhal (estenose espinhal) e a formação de cistos preenchidos por líquido (siringe) dentro da medula espinhal.

Com tratamento precoce, pessoas com hipofosfatemia ligada ao X podem apresentar sinais e sintomas esqueléticos menos graves e menos problemas de saúde a longo prazo.

Fonte: MedlinePlus Genetics (NLM/NIH), traduzido
📋
Informacoes curadas por IA — podem conter imprecisoes

O raquitismo hipofosfatêmico é um grupo de doenças genéticas caracterizadas por hipofosfatemia, raquitismo e níveis séricos normais de cálcio.

Pesquisas ativas
10 ensaios
63 total registrados no ClinicalTrials.gov
Publicações científicas
1.377 artigos
Último publicado: 2026
Medicamentos
1 com registro
Burosumabe

Tem tratamento?

✓ 1 medicamento específico desta doença (registro ANVISA, FDA ou SUS/CEAF)
Ver detalhes, fases e interações →
BUROSUMABE

Escala de raridade

CLASSIFICAÇÃO ORPHANET · BRASIL 2024
Unknown
Ultra-rara
<1/50k
Muito rara
1/20k
Rara
1/10k
Pouco freq.
1/5k
Incomum
1/2k
Prevalência
0.0
Worldwide
Início
All ages
🏥
SUS: Sem cobertura SUSScore: 0%
1 medicamento CEAFCID-10: E83.31
Você se identifica com essa condição?
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Entender a doença

Do básico ao detalhe, leia no seu ritmo

Preparando trilha educativa...

Sinais e sintomas

O que aparece no corpo e com que frequência cada sintoma acontece

Partes do corpo afetadas

🦴
Ossos e articulações
43 sintomas
🫘
Rins
12 sintomas
📏
Crescimento
8 sintomas
🦷
Dentes
6 sintomas
🫃
Digestivo
3 sintomas
👂
Ouvidos
2 sintomas

+ 52 sintomas em outras categorias

Características mais comuns

—
Achatamento da cúpula talar
—
Aumento do nível circulante de osteocalcina
—
Aumento do nível circulante de telopeptídeo beta-C-terminal
—
Fratura óssea
—
Disfagia
—
Limitação da mobilidade articular
131sintomas
Sem dados (131)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 131 características clínicas mais associadas, ordenadas por frequência.

Achatamento da cúpula talarFlattening of the talar dome
Aumento do nível circulante de osteocalcinaIncreased circulating osteocalcin level
Aumento do nível circulante de telopeptídeo beta-C-terminalIncreased circulating beta-C-terminal telopeptide level
Fratura ósseaBone fracture
DisfagiaDysphagia

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa1desde 2026
Total histórico1.377PubMed
Últimos 10 anos200publicações
Pico2025112 papers
Linha do tempo
2026Hoje · 2026🧪 2004Primeiro ensaio clínico📈 2025Ano de pico
Publicações por ano (últimos 10 anos)

Encontrou um erro ou informação desatualizada? Sugira uma correção →

Genética e causas

O que está alterado no DNA e como passa nas famílias

Genes associados

7 genes identificados com associação a esta condição. Padrão de herança: Autosomal dominant, Autosomal recessive, X-linked dominant.

DMP1Dentin matrix acidic phosphoprotein 1Disease-causing germline mutation(s) inTolerante
VIAS BIOLÓGICAS (2)
Post-translational protein phosphorylationRegulation of Insulin-like Growth Factor (IGF) transport and uptake by Insulin-like Growth Factor Binding Proteins (IGFBPs)
EXPRESSÃO TECIDUAL(Baixa expressão)
Testículo
1.9 TPM
Aorta
0.2 TPM
Artéria tibial
0.2 TPM
Artéria coronária
0.1 TPM
Pituitária
0.1 TPM
OUTRAS DOENÇAS (2)
hypophosphatemic rickets, autosomal recessive, 1autosomal recessive hypophosphatemic rickets
HGNC:2932UniProt:Q13316
SLC34A1Sodium-dependent phosphate transport protein 2ACandidate gene tested inTolerante
VIAS BIOLÓGICAS (2)
Surfactant metabolismType II Na+/Pi cotransporters
MECANISMO DE DOENÇA

Nephrolithiasis/osteoporosis, hypophosphatemic, 1

A disease characterized by decreased renal phosphate absorption, renal phosphate wasting, hypophosphatemia, hyperphosphaturia, hypercalciuria, nephrolithiasis and osteoporosis.

EXPRESSÃO TECIDUAL(Tecido-específico)
Rim - Córtex
35.5 TPM
Rim - Medula
19.9 TPM
Fígado
0.6 TPM
Esôfago - Mucosa
0.3 TPM
Skin Not Sun Exposed Suprapubic
0.2 TPM
OUTRAS DOENÇAS (7)
hypophosphatemic nephrolithiasis/osteoporosis 1hypercalcemia, infantile, 2Fanconi renotubular syndrome 2obsolete dominant hypophosphatemia with nephrolithiasis or osteoporosis
HGNC:11019UniProt:Q06495
SLC34A3Sodium-dependent phosphate transport protein 2CDisease-causing germline mutation(s) inTolerante
VIAS BIOLÓGICAS (1)
Type II Na+/Pi cotransporters
MECANISMO DE DOENÇA

Hereditary hypophosphatemic rickets with hypercalciuria

Autosomal recessive form of hypophosphatemia characterized by reduced renal phosphate reabsorption and rickets. Increased serum levels of 1,25-dihydroxyvitamin D lead to increase in urinary calcium excretion.

EXPRESSÃO TECIDUAL(Tecido-específico)
Rim - Córtex
9.1 TPM
Pituitária
4.6 TPM
Esôfago - Mucosa
3.8 TPM
Rim - Medula
2.8 TPM
Testículo
1.4 TPM
OUTRAS DOENÇAS (1)
hereditary hypophosphatemic rickets with hypercalciuria
HGNC:20305UniProt:Q8N130
PHEXPhosphate-regulating neutral endopeptidase PHEXDisease-causing germline mutation(s) inAltamente restrito
MECANISMO DE DOENÇA

Hypophosphatemic rickets, X-linked dominant

A disorder characterized by impaired phosphate uptake in the kidney, which is likely to be caused by abnormal regulation of sodium phosphate cotransport in the proximal tubules. Clinical manifestations include skeletal deformities, growth failure, craniosynostosis, paravertebral calcifications, pseudofractures in lower extremities, and muscular hypotonia with onset in early childhood. X-linked hypophosphatemic rickets is the most common form of hypophosphatemia with an incidence of 1 in 20000.

EXPRESSÃO TECIDUAL(Tecido-específico)
Pulmão
5.1 TPM
Útero
3.6 TPM
Brain Spinal cord cervical c-1
2.1 TPM
Cervix Endocervix
2.0 TPM
Tireoide
1.6 TPM
OUTRAS DOENÇAS (1)
X-linked dominant hypophosphatemic rickets
HGNC:8918UniProt:P78562
ENPP1Ectonucleotide pyrophosphatase/phosphodiesterase family member 1Disease-causing germline mutation(s) inTolerante
VIAS BIOLÓGICAS (2)
Vitamin B5 (pantothenate) metabolismVitamin B2 (riboflavin) metabolism
MECANISMO DE DOENÇA

Ossification of the posterior longitudinal ligament of the spine

A calcification of the posterior longitudinal ligament of the spinal column, usually at the level of the cervical spine. Patients with OPLL frequently present with a severe myelopathy that can lead to tetraparesis.

EXPRESSÃO TECIDUAL(Ubíquo)
Útero
42.3 TPM
Tireoide
26.5 TPM
Aorta
22.2 TPM
Pituitária
22.0 TPM
Fígado
19.8 TPM
OUTRAS DOENÇAS (8)
hypophosphatemic rickets, autosomal recessive, 2hypopigmentation-punctate palmoplantar keratoderma syndromearterial calcification, generalized, of infancy, 1autosomal recessive inherited pseudoxanthoma elasticum
HGNC:3356UniProt:P22413
FGF23Fibroblast growth factor 23Disease-causing germline mutation(s) inTolerante
VIAS BIOLÓGICAS (2)
Post-translational protein phosphorylationRegulation of Insulin-like Growth Factor (IGF) transport and uptake by Insulin-like Growth Factor Binding Proteins (IGFBPs)
MECANISMO DE DOENÇA

Hypophosphatemic rickets, autosomal dominant

A disease characterized by isolated renal phosphate wasting, hypophosphatemia, and inappropriately normal 1,25-dihydroxyvitamin D3 (calcitriol) levels. Patients frequently present with bone pain, rickets, and tooth abscesses.

EXPRESSÃO TECIDUAL(Baixa expressão)
Coração - Átrio
0.8 TPM
Fígado
0.2 TPM
Testículo
0.1 TPM
Coração - Ventrículo esquerdo
0.0 TPM
Intestino delgado
0.0 TPM
OUTRAS DOENÇAS (3)
tumoral calcinosis, hyperphosphatemic, familial, 2autosomal dominant hypophosphatemic ricketsfamilial hyperphosphatemic tumoral calcinosis/hyperphosphatemic hyperostosis syndrome
HGNC:3680UniProt:Q9GZV9
CLCN5H(+)/Cl(-) exchange transporter 5Disease-causing germline mutation(s) inAltamente restrito
VIAS BIOLÓGICAS (1)
Stimuli-sensing channels
MECANISMO DE DOENÇA

Hypophosphatemic rickets, X-linked recessive

A renal disease belonging to the 'Dent disease complex', a group of disorders characterized by proximal renal tubular defect, hypercalciuria, nephrocalcinosis, and renal insufficiency. The spectrum of phenotypic features is remarkably similar in the various disorders, except for differences in the severity of bone deformities and renal impairment. XLHRR patients present with rickets or osteomalacia, hypophosphatemia due to decreased renal tubular phosphate reabsorption, hypercalciuria, and low molecular weight proteinuria. Patients develop nephrocalcinosis with progressive renal failure in adulthood. Female carriers may have asymptomatic hypercalciuria or hypophosphatemia only.

VIAS REACTOME (1)
OUTRAS DOENÇAS (4)
hypophosphatemic rickets, X-linked recessivenephrolithiasis, X-linked recessive, with renal failureproteinuria, low molecular weight, with hypercalciuria and nephrocalcinosisDent disease type 1
HGNC:2023UniProt:P51795

Variantes genéticas (ClinVar)

482 variantes patogênicas registradas no ClinVar.

🧬 DMP1: NM_004407.4(DMP1):c.54+1G>A ()
🧬 DMP1: NM_004407.4(DMP1):c.273del (p.Ser92fs) ()
🧬 DMP1: NM_004407.4(DMP1):c.534del (p.Glu179fs) ()
🧬 DMP1: NM_004407.4(DMP1):c.55-2A>T ()
🧬 DMP1: NM_004407.4(DMP1):c.683dup (p.Asn229fs) ()
Ver todas no ClinVar

Classificação de variantes (ClinVar)

Distribuição de 662 variantes classificadas pelo ClinVar.

364
265
33
Patogênica (55.0%)
VUS (40.0%)
Benigna (5.0%)
VARIANTES MAIS SIGNIFICATIVAS
CLCN5: NM_001127898.4(CLCN5):c.603+1G>T [Pathogenic]
CLCN5: NM_001127898.4(CLCN5):c.1975del (p.Arg659fs) [Likely pathogenic]
CLCN5: NM_001127898.4(CLCN5):c.1799G>T (p.Gly600Val) [Pathogenic]
CLCN5: NM_001127898.4(CLCN5):c.1726G>A (p.Gly576Arg) [Pathogenic]
CLCN5: NM_001127898.4(CLCN5):c.1616T>G (p.Leu539Arg) [Likely pathogenic]

Vias biológicas (Reactome)

42 vias biológicas associadas aos genes desta condição.

Diagnóstico

Os sinais que médicos procuram e os exames que confirmam

Carregando...

Tratamento e manejo

Remédios, cuidados de apoio e o que precisa acompanhar

Pipeline de tratamentos
Pipeline regulatório — de medicamentos já aprovados a drogas em pesquisa exploratória.
✓Aprovado5
3Fase 312
2Fase 211
1Fase 16
·Pré-clínico29
Medicamentos catalogadosEnsaios clínicos· 0 medicamentos · 63 ensaios
Carregando informações de tratamento...

Onde tratar no SUS

Hospitais de referência no Brasil e o protocolo oficial do SUS (PCDT)

🇧🇷 Atendimento SUS — Raquitismo hipofosfatêmico

🗺️

Selecione um estado ou use sua localização para ver resultados.

Dados de DATASUS/CNES, SBGM, ABNeuro e Ministério da Saúde. Sempre confirme a disponibilidade diretamente com o estabelecimento.

Pesquisa ativa

Ensaios clínicos abertos e novidades científicas recentes

Onde estão os ensaios

Com ensaio aberto em 8 países. O ponto verde marca onde há vaga agora.

🟢 Recrutando agora

4 pesquisas recrutando participantes. Converse com seu médico sobre a possibilidade de participar.

Outros ensaios clínicos

63 ensaios clínicos encontrados, 10 ativos.

Distribuição por fase
NCT07666269 · Morphology in Oral Rare Syndromes & Artificial Intelligence …Em breve
NA🇫🇷 França
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🇧🇷 Brasil · 🇦🇷 Argentina · 🇨🇦 Canadá +3
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NCT04946409 · Burden of Disease and Functional Impairment in XLHAtivo
🇩🇪 Alemanha
NCT03745521 · Study of Longitudinal Observation for Patient With X-linked …Ativo
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NCT05050669 · Natural History Study of ENPP1 Deficiency and the Early-onse…Concluído
🇨🇦 Canadá · 🇬🇧 Reino Unido · 🇺🇸 Estados Unidos
NCT05181839 · A Study to Describe the Lived Experience of XLH for Adolesce…Concluído
🇫🇷 França · 🇩🇪 Alemanha · 🇳🇱 Países Baixos +2
NCT04686175 · Evaluation of Safety, Tolerability, and Efficacy of INZ-701 …Concluído
PHASE1, PHASE2🇨🇦 Canadá · 🇫🇷 França · 🇩🇪 Alemanha +2
NCT04842032 · Study to Assess the Safety, Pharmacokinetics and Efficacy of…Concluído
PHASE4🇨🇳 China
NCT04842019 · Study to Assess the Safety, Pharmacokinetics and Efficacy of…Concluído
PHASE4🇨🇳 China
NCT04819490 · A UK Multicentre, Health-Related Quality of Life Study for C…Concluído
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NCT04184661 · Burosumab and 1-25 (OH) Vitamin D on Human OsteoclastsConcluído
🇫🇷 França
NCT04695860 · Anti-FGF23 (Burosumab) in Adult Patients With XLHConcluído
PHASE3🇩🇪 Alemanha
NCT06067932 · Foot Disorders in X-linked HypophosphatemiaConcluído
🇦🇹 Áustria
NCT04372446 · Understanding the Spectrum of ENPP1 Deficiency and Acute ABC…Concluído
🇺🇸 Estados Unidos
NCT04188964 · Study to Assess the Safety, Tolerability, Pharmacokinetics a…Concluído
PHASE1, PHASE2🇦🇹 Áustria · 🇫🇷 França · 🇮🇹 Itália +3
NCT04273490 · Characterising Pain, QoL, Body Composition, Arterial Stiffne…Concluído
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NCT04146935 · Examining the Effect of Burosumab on Muscle FunctionConcluído
PHASE4🇺🇸 Estados Unidos
NCT04049877 · Retrospective and Prospective Disease Progression and Qualit…Concluído
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NCT03748966 · Calcitriol Monotherapy for X-Linked HypophosphatemiaConcluído
EARLY_PHASE1🇺🇸 Estados Unidos
NCT03920072 · Study of the Anti-FGF23 Antibody, Burosumab, in Adults With …Concluído
PHASE3🇫🇷 França · 🇮🇪 Irlanda · 🇮🇹 Itália +1
NCT03596554 · X-linked Hypophosphatemia and FGF21Concluído
🇫🇷 França
NCT03489993 · FGF23 and Angiotensin-(1-7) in Hypophosphatemia (GAP)Concluído
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NCT07745179 · Natural History Study: ENPP1 Deficiency or the Early-Onset F…Concluído
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NCT03478839 · Study of People With Generalized Arterial Calcification of I…Concluído
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NCT03581591 · Open Label Trial Assessing Safety and Efficacy of Burosumab …Concluído
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NCT03233126 · A Study of KRN23 in Pediatric Patients With X-linked Hypopho…Concluído
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NCT02526160 · Study of KRN23 in Adults With X-linked Hypophosphatemia (XLH…Concluído
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NCT02312687 · Long-Term Extension Study of KRN23 in Adult Subjects With X-…Concluído
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NCT02233322 · Iron Therapy for Autosomal Dominant Hypophosphatemic Rickets…Concluído
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PHASE1, PHASE2🇨🇦 Canadá · 🇺🇸 Estados Unidos
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NCT01237288 · Therapeutic Use of Oral Sodium Phosphate (Z-521) in Primary …Concluído
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NCT00830674 · A Study of KRN23 in X-linked HypophosphatemiaConcluído
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NCT00417612 · Effectiveness of Paricalcitol in Reducing Parathyroid Hormon…Concluído
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NCT03758534 · Natural History of GACI With or Without ARHR2 or PXEUNKNOWN
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PHASE3🇦🇺 Austrália · 🇨🇦 Canadá · 🇫🇷 França +5
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Ver todos no ClinicalTrials.gov
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Publicações mais relevantes

🥇Melhor nível de evidência: Revisão sistemática
Timeline de publicações
602 papers (10 anos)

Mostrando amostra de 200 publicações de um total de 602

#1

The lived experience of adolescents with X-linked hypophosphataemia treated with burosumab at end of skeletal growth: a mixed-methods analysis.

PloS one2026

X-linked hypophosphataemia is a rare, genetic, lifelong disorder caused by phosphate-regulating endopeptidase homologue X-linked pathogenic variants and, if left untreated, is associated with a progressive accumulation of musculoskeletal manifestations. Burosumab is a fully human monoclonal antibody that targets circulating fibroblast growth factor 23 and directly inhibits its activity, thereby correcting the abnormal phosphate homoeostasis in people with X-linked hypophosphataemia (XLH). The efficacy and safety of burosumab has been demonstrated in a programme of clinical trials in children and adults. Few data describe the experience of adolescents with XLH receiving burosumab treatment before and after skeletal growth ends. This prospective, multicentre, mixed-methods study described the lived experience of adolescents with XLH treated with burosumab at the end of skeletal growth (NCT05181839). Using patient-reported outcomes, wearable devices, and interviews, we found low median symptom severity scores for pain (0.00), stiffness (0.00), and fatigue (1.75) on a 0-10 scale. Symptoms were usually triggered by physical activity but rarely interfered with daily life. Some adolescents reported emotional concerns related to XLH and treatment transition. These insights can inform patient support during transition to adult care.

#2

Composite Phenotype: Recurrent Nephrolithiasis and Chronic Kidney Disease in an Adult with Biallelic SLC34A3 and Monoallelic SLC3A1 Pathogenic Variants: Who is 'The Culprit'?

Kidney &amp; blood pressure research2026 Mar 18

Kidney stones are common and can arise from many etiologies including genetic and environmental. Biallelic pathogenic variants in the solute carrier family 34-member 3 (SLC34A3) gene cause Hereditary Hypophosphatemic Rickets with Hypercalciuria (HHRH), while both monallaelic and biallelic pathogenic variants in SLC3A1 cause cystinuria. Here, we report the clinical phenotype of a patient with concomitant biallelic and monoallelic pathogenic variants in SLC34A3 and SLC3A1 respectively.

#3

Refractory Rickets: Evaluation and Management.

Indian journal of pediatrics2026 Feb 26

Refractory rickets refers to a set of diseases which are identified by a lack of response to therapeutic doses used to treat vitamin D deficiency. A child presenting with refractory rickets can pose a diagnostic dilemma as many kidney diseases have been identified as possible causes. Inherited (e.g., distal renal tubular acidosis, Fanconi syndrome, hypophosphatemic rickets, vitamin D-dependent rickets, nephronophthisis) and acquired tubular disorders [e.g., posterior urethral valves, reflux nephropathy leading to chronic kidney disease (CKD)-mineral bone disorder] may be complicated by refractory rickets. Rarely, chronic liver disease and malabsorption states can also result in refractory rickets. Hypophosphatemia is a feature of both calcipenic as well as phosphopenic rickets. Common features accompanying refractory rickets include polyuria, polydipsia, hypokalemic paralysis, fractures, limb deformities, failure-to-thrive, short-stature, tetany and nephrocalcinosis. A careful history, examination and biochemical evaluation is required to delineate the underlying cause. Using a rational algorithmic approach, it is possible to determine the correct diagnosis in these cases. Consequent upon easy availability of next generation sequencing (NGS), the accurate diagnoses can be promptly made aiding in targeted therapy. Children with refractory rickets need regular follow-up to optimise the biochemical abnormalities, monitor growth and retard the progression of CKD. This article describes the evaluation of a child with refractory rickets using an algorithmic approach, underscores the importance of the necessary blood and urine biochemical tests as well as NGS for identification of the precise etiology of refractory-rickets; and discusses the pathophysiology and management of the most important causes of refractory-rickets.

#4

Burosumab in infants with X-linked hypophosphatemic rickets: a case series.

Orphanet journal of rare diseases2026 Jan 08

X-linked hypophosphatemic rickets (XLH) is a rare inherited metabolic bone disorder caused by excess fibroblast growth factor 23 (FGF23), leading to hypophosphatemia and rickets. Burosumab, a human monoclonal antibody targeting FGF23, was approved for the treatment of XLH in April 2018. By 2022, the FDA extended its approval to include children as young as six months of age. To describe three infants with XLH who began burosumab therapy before one year of age and were monitored for at least one year. Case series. Clinical outcomes, including anthropometric measures, skeletal outcomes (Rickets Severity Score [RSS], mechanical axis deviation [MAD], and neck-shaft angle [NSA]), and laboratory parameters, were assessed in a real-world setting. Two patients demonstrated satisfactory linear growth, and one experienced growth faltering, possibly due to sleep-disordered breathing or phosphate imbalance. These patients received higher doses of burosumab than the current guideline recommendations for achieving treatment goals aimed at normalizing phosphate levels. The patient’s phosphate levels improved but did not normalize. Bone pain was not formally assessed, but parents reported improvements in their children’s conditions. Importantly, two patients with assessable mechanical axes demonstrated neutral mechanical axis deviations, indicating improved lower limb alignment and supporting the therapeutic efficacy of burosumab. All three patients had favorable RSS outcomes, and none developed long bone diaphyseal bowing or coxa vara following this early initiation of burosumab treatment. This case series demonstrated potential benefits of early initiation of burosumab treatment for XLH by showing improvements in growth, phosphate levels, and skeletal outcomes. Burosumab appears well tolerated in infancy, but further research is needed to refine dosing strategies and assess its long-term safety and therapeutic efficacy in young patients with XLH. Meticulous monitoring and individualized care are essential throughout treatment. This case series followed three infants with X-linked hypophosphatemic rickets (XLH) who initiated burosumab treatment at 6 months of age, filling a significant knowledge gap in early intervention. Despite requiring higher-than-recommended dosing, burosumab demonstrated favorable outcomes in terms of growth parameters and skeletal development over a minimum one-year follow-up. Two patients maintained normal growth trajectories, whereas one experienced temporary growth faltering, which resolved after adenoidectomy. Importantly, neutral mechanical axis deviation was achieved, indicating effective prevention of lower limb deformities. No significant adverse effects were observed. Despite limitations such as a small sample size and short follow-up, these findings suggest that early burosumab therapy can improve outcomes in infantile XLH patients. Further research is needed to refine dosing protocols and assess long-term safety and efficacy in this young population.

#5

Treatment with Minicircle DNA Expressing a FGF23 Fragment in a Clinically relevant Mouse Model of X-Linked Hypophosphatemic Rickets.

Advanced science (Weinheim, Baden-Wurttemberg, Germany)2026 Jan

X-linked hypophosphatemic rickets (XLHR) is a rare X-linked dominant skeletal dysplasia caused by phosphate regulating endopeptidase homolog X-linked (PHEX) gene mutation. Until now, the pathogenic role of PHEX has not been fully determined, and there has been no radical cure for XLHR. In the previous study, a novel PHEX variant (c.T1349C; p.L450P) is identified in a child with XLHR. The present study aims to reveal its pathogenic role and evaluate the therapeutic effects of the minicircle (MC)-DNA in XLHR. In vitro, the wildtype and mutant plasmids are introduced into HEK293 cells. In vivo, a new knock-in XLHR mouse model carrying the novel variant is established. Furthermore, this study makes the first attempt to perform gene therapy using a MC-DNA vector expressing a fragment of FGF23 (amino acids 180-251) in the Phex-T1349C mice. The new mouse model demonstrates the clinical manifestations of XLHR seen in the patient, including a gene dosage effect. Furthermore, MC-DNA is found to slightly increase blood phosphorus levels, significantly decrease serum alkaline phosphatase levels, and improve bone mineralization without apparent adverse effects for at least 6 weeks. This study suggests MC-DNA as a promisingly safe and effective therapeutic strategy to treat XLHR.

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2026

Reference intervals for intact FGF 23 in healthy Korean adults: lower concentrations in young adulthood require age-specific partitioning.

Frontiers in endocrinology
2026

The lived experience of adolescents with X-linked hypophosphataemia treated with burosumab at end of skeletal growth: a mixed-methods analysis.

PloS one
2026

Composite Phenotype: Recurrent Nephrolithiasis and Chronic Kidney Disease in an Adult with Biallelic SLC34A3 and Monoallelic SLC3A1 Pathogenic Variants: Who is 'The Culprit'?

Kidney &amp; blood pressure research
2026

A Japanese Patient With Craniosynostosis Due to X-Linked Hypophosphatemic Rickets Who Underwent Fronto-Orbital Distraction.

The Journal of craniofacial surgery
2026

Expanding the Autosomal Recessive Hypophosphatemic Rickets Type I Carrier Phenotype and Adult Treatment with Burosumab.

Calcified tissue international
2026

Unveiling the unexpected: refractory rickets as an uncommon presentation of tyrosinemia type I.

BMC pediatrics
2026

Optimizing diagnosis and management of patients with ENPP1 deficiency: an expert opinion.

Journal of endocrinological investigation
2025

X-linked hypophosphatemic rickets: a rare case report.

Acta orthopaedica Belgica
2026

Refractory Rickets: Evaluation and Management.

Indian journal of pediatrics
2026

Transition of Care in X-Linked Hypophosphatemic Rickets: From Pediatric to Adult Practice- A Narrative Review.

Journal of clinical research in pediatric endocrinology
2026

Autoimmune osteomalacia: a novel FGF23-related hypophosphatemic osteomalacia.

Journal of bone and mineral metabolism
2026

Phenotypic Diversity in Autosomal Recessive Hypophosphatemic Rickets Type 2.

Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research
2026

Current advances of bone homeostasis imbalance in the cause of hereditary metabolic bone diseases.

EFORT open reviews
2025

Early Dental Manifestations and Multidisciplinary Management of X-Linked Hypophosphatemic Rickets in a Pediatric Patient: A Case Report.

Children (Basel, Switzerland)
2026

[Metabolic bone diseases : what's new in 2025].

Revue medicale suisse
2026

Heritable metabolic bone disorders: a guide to current genetic testing and clinical management for adult endocrinologists.

Pathology
2026

Burosumab in infants with X-linked hypophosphatemic rickets: a case series.

Orphanet journal of rare diseases
2025

Biochemical evaluation of X-linked hypophosphatemia and tumor-induced osteomalacia: insights into diagnosis and management.

Frontiers in endocrinology
2026

Dental disease burden and management in children and young people with X-linked hypophosphataemia: a scoping review and service evaluation.

European archives of paediatric dentistry : official journal of the European Academy of Paediatric Dentistry
2025

Six cases of ENPP1 pathogenic variants causing autosomal recessive hypophosphatemic rickets type 2 and generalized arterial calcification of infancy.

JBMR plus
2025

A case and review of fibroblast growth factor-23-mediated hypophosphatemic osteomalacia in the absence of pathogenic PHEX variants.

JBMR plus
2025

Patient and carer perceptions and acceptability of current management practices in paediatric X-linked hypophosphatemia treated with burosumab therapy.

JBMR plus
2025

Burosumab in adults with X-linked hypophosphatemia: real-world experience from a retrospective study in Sydney.

JBMR plus
2026

X-linked hypophosphatemia in childhood: dental involvement, diagnosis, and treatment.

European journal of paediatric dentistry
2025

Lower Limb Deformity in Different Types of Rickets.

Journal of clinical medicine
2026

Characterization of monogenic diabetes among Sudanese children: a multi-center experience from a population with high consanguinity.

Journal of pediatric endocrinology &amp; metabolism : JPEM
2025

Pediatric ABCC6 deficiency: a genotypic and phenotypic analysis.

Orphanet journal of rare diseases
2025

A bibliometric approach to worldwide scientific production of familial hypophosphataemic rickets in Scopus (2000-2022).

Orphanet journal of rare diseases
2025

The prevalence of ENPP1 deficiency in humans with OPLL and the preclinical efficacy of ENPP1 enzyme therapy in OPLL mice.

Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research
2025

FAM20C and FAM20A in normal and ectopic mineralization: A focus on oro-renal syndromes.

Matrix biology : journal of the International Society for Matrix Biology
2026

Secondary Deformity After Hemiepiphysiodesis in Hypophosphatemic Rickets: The Role of Metabolic Control.

Journal of pediatric orthopedics
2025

Case Report: Increased FGF23 and new insufficiency fractures at burosumab discontinuation in X-linked hypophosphatemia.

Frontiers in endocrinology
2026

Autosomal recessive hypophosphatemic rickets type 2 (ARHR2): Is phosphate supplementation safe?

Bone
2026

Treatment with Minicircle DNA Expressing a FGF23 Fragment in a Clinically relevant Mouse Model of X-Linked Hypophosphatemic Rickets.

Advanced science (Weinheim, Baden-Wurttemberg, Germany)
2025

A Rare Cause of Sacral Insufficiency Fracture in Adolescence: Autosomal Dominant Hypophosphatemic Rickets due to Fgf23 de novo P.Arg176trp Variant.

Journal of clinical research in pediatric endocrinology
2026

Treatment of Children and Adults With X-Linked Hypophosphatemia With Calcitriol Alone: A Prospective, Open-Label Study.

The Journal of clinical endocrinology and metabolism
2025

Deformity Correction in an Adult With Hypophosphatemic Rickets.

Cureus
2025

Feline vitamin D-dependent rickets type 2 caused by a missense variant in the vitamin D receptor gene.

The Journal of veterinary medical science
2026

Surgical and Nonsurgical Endodontic Treatment of a Patient with Vitamin D-resistant Rickets and Extraoral Sinus Tract: A Case Report.

Journal of endodontics
2025

A case of autosomal dominant hypophosphatemic rickets.

Clinical pediatric endocrinology : case reports and clinical investigations : official journal of the Japanese Society for Pediatric Endocrinology
2026

Distribution of blood pressure and its positive association with body mass index standard deviation score in pediatric patients with X-linked hypophosphatemia: a sub-group analysis from the SUNFLOWER observational study.

Endocrine journal
2026

A deep intronic PHEX variant in a large Danish family with hereditary hypophosphatemia and a milder skeletal, but more severe dental phenotype.

Bone
2025

Effect of burosumab conversion on calciuria and nephrocalcinosis in children with XLH: A real-world cohort study.

Bone reports
2025

Burosumab prevents further height deficit in toddlers affected by XLH.

Endocrine connections
2025

Variants of unknown significance are common in brushite stone formers undergoing genetic testing for nephrolithiasis.

Urolithiasis
2025

A novel phex gene variant causes non-syndromic tooth agenesis.

BMC oral health
2025

Concordant X-linked hypophosphatemic rickets in monozygotic twins: diagnostic challenges and a novel genetic insight.

Endocrinology, diabetes &amp; metabolism case reports
2025

Improvement in Physical Function Associated With Burosumab in a Patient With X-linked Hypophosphatemia: A Case Report.

Cureus
2025

Real-World Effectiveness of Burosumab in Adults with X-Linked Hypophosphataemia (XLH) in the UK.

Calcified tissue international
2025

Advancing Patient Evidence in XLH (APEX): Baseline analysis of a global data unification program.

Bone
2025

Clinical and Laboratory Characteristics of Patients with Genetic Rickets: A Single-Center Experience with Long-Term Outcomes.

Turkish archives of pediatrics
2025

Periodontal Manifestations of Systemic Diseases.

Journal of periodontal research
2025

Identification of a Novel Homozygous SLC34A1 Missense Mutation and a Heterozygous SLC34A3 Deletion in an Infant with Nephrocalcinosis, Failure to Thrive, and Hypercalcemia.

International journal of molecular sciences
2025

X-Linked Hypophosphatemia: Role of Fibroblast Growth Factor 23 on Human Skeletal Muscle-Derived Cells.

Calcified tissue international
2025

Clinical Characteristics and Management of Rare Metabolic Bone Diseases: An Audit of the Rare Metabolic Bone Disease Registry of India.

Calcified tissue international
2025

Oral Health Status and Parental Awareness in Children with X-Linked Hypophosphatemic Rickets: A Case-Control Study.

Reports (MDPI)
2026

Challenges in Estimating Renal Function in X-linked Hypophosphatemia Because of Formula Overestimation and FGF23 Effects.

The Journal of clinical endocrinology and metabolism
2025

Lower limb deformity correction in X-linked hypophosphatemia (XLH) with intramedullary nails: A strategy to reduce deformity recurrence and improve limb function.

Bone
2025

Hereditary Hypophosphatemic Rickets with Hypercalciuria - Importance of Further Evaluation If Clinical Suspicion is Strong.

Journal of clinical research in pediatric endocrinology
2025

A case report of X-linked hypophosphatemia combined with primary hyperparathyroidism.

Frontiers in endocrinology
2025

3D-printed model combined with the Ilizarov technique in the treatment of genu varum in children with hypophosphatemic rickets.

Journal of orthopaedic surgery and research
2025

[Delayed bone healing after osteotomy - what the family history revealed].

Praxis
2025

The genetic polymorphism of XPR1 associated with Fanconi syndrome in Chinese patients with X-linked hypophosphatemia.

Journal of endocrinological investigation
2025

A Novel PHEX Mutation in X-Linked Hypophosphataemic Rickets With Reduced Expression of NaPi-IIa and NaPi-IIc in the Proximal Tubules: A Case Report.

Nephrology (Carlton, Vic.)
2025

Autosomal dominant hypophosphatemic rickets: a case report of two sisters with a novel FGF-23 mutation.

Osteoporosis international : a journal established as result of cooperation between the European Foundation for Osteoporosis and the National Osteoporosis Foundation of the USA
2025

Unexpected SLC34A3 rickets in a case of suspected distal myopathy.

Pediatric nephrology (Berlin, Germany)
2025

Prevalence of low birth weight, short length, and body disproportion at birth in patients with skeletal dysplasias: A retrospective study.

Archivos argentinos de pediatria
2025

3D-DXA reveals significant effects of burosumab on trabecular and cortical skeletal envelopes in symptomatic adults with X-linked hypophosphatemia.

Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research
2025

Iron infusion in pregnancy and dental dysplasia in children-is there a link?

Frontiers in pediatrics
2026

Pamidronate Treatment of a Patient with Opsismodysplasia and a Novel INPPL1 Variant: Efficacy, Mechanism, and Clinical Outcomes.

Molecular syndromology
2025

Targeted treatment of hypophosphatemia with trametinib in HRAS-related mosaic RASopathy.

Orphanet journal of rare diseases
2025

Clustered Structural Variants Involving <italic>PHEX</italic> at Xp22 in a Female Patient with X-Linked Hypophosphatemia.

Cytogenetic and genome research
2025

Experts' consensus on the management and treatment of individuals with X-linked hypophosphatemia across lifespan.

Journal of endocrinological investigation
2025

[Surgical strategies for osteotomy correction of severe lower limb deformities in hypophosphatemic rickets].

Zhongguo xiu fu chong jian wai ke za zhi = Zhongguo xiufu chongjian waike zazhi = Chinese journal of reparative and reconstructive surgery
2025

First case of preimplantation genetic testing of X-linked dominantly inherited hypophosphatemia family lines using next-generation sequencing technology.

European journal of obstetrics, gynecology, and reproductive biology
2025

Extracellular Matrix Proximity Biotinylation Identifies Periostin as a PHEX Proteolytic Substrate.

FASEB journal : official publication of the Federation of American Societies for Experimental Biology
2025

A Mosaic PHEX Variant in Hypophosphatemic Rickets: Distinguishing Postzygotic Mutation from Sex Chromosome Aneuploidy.

Calcified tissue international
2025

PHEX Protein in the Parathyroid Gland Contributes to Phosphate Sensing.

The Journal of clinical endocrinology and metabolism
2025

Switching from active vitamin D and phosphate supplementation to burosumab significantly corrects lower limb malalignment in pediatric X-linked hypophosphatemia.

Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research
2025

Improvements in hearing loss with bone-targeted enzyme replacement therapy are associated with corrected hypomineralization and osteocyte properties of auditory ossicles in Enpp1-deficient mice.

Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research
2025

Growth dynamics of transversal body dimensions and proportions, with related clinical determinants in children with X-linked hypophosphatemia treated with phosphate supplements and active vitamin D.

Pediatric nephrology (Berlin, Germany)
2025

Inadequate pediatric reference ranges impede the diagnosis of X-linked hypophosphatemia and hypophosphatasia in Austria.

Wiener klinische Wochenschrift
2025

Real-world characteristics & disease history of patients with X-linked hypophosphatemia before treatment with burosumab.

Archives of osteoporosis
2025

Oncogenic rickets diagnosed at age 8 and the risk of persistent rickets: a rare case of pediatric-onset tumor-induced osteomalacia.

Osteoporosis international : a journal established as result of cooperation between the European Foundation for Osteoporosis and the National Osteoporosis Foundation of the USA
2025

Predictors of response to burosumab in adults with X-linked hypophosphatemia: real-world data from an Italian cohort.

Journal of endocrinological investigation
2025

Real-world effectiveness of burosumab vs oral phosphate and active vitamin D in adults with X-linked hypophosphatemia.

Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research
2025

Quality of life of 26 family members from four generations with X-linked hypophosphatemia: a cross-sectional study.

Frontiers in endocrinology
2025

A Child with Cutaneous-Skeletal Hypophosphatemia Syndrome Caused by a Mosaic HRAS Mutation: Outcome of Treatment with Anti-FGF23 Antibody.

Calcified tissue international
2025

The Diagnosis and Therapy of XLH.

Calcified tissue international
2025

The impact of monoallelic inactivation mutations in the ENPP1 gene on pediatric skeletal development: a case report and literature review.

Frontiers in endocrinology
2025

Advancing patient evidence in XLH (APEX): rationale and design of a real-world XLH global data unification program.

Frontiers in endocrinology
2025

X-Linked Hypophosphatemia Management in Adults: An International Working Group Clinical Practice Guideline.

The Journal of clinical endocrinology and metabolism
2025

Case report: Reversible Fanconi syndrome due to vitamin D deficiency in a patient with epilepsy harbouring a pathogenic variant in the SLC34A1 gene.

Frontiers in endocrinology
2025

Craniosynostosis among children with X-linked hypophosphatemia: A systematic review and meta-analysis.

Bone
2025

Vitamin D dependent rickets type 2A: a case series of two siblings with novel mutation in vitamin D receptor gene responded to high dose oral calcium and calcitriol.

Journal of pediatric endocrinology &amp; metabolism : JPEM
2025

Characterization of adult patients with X-linked hypophosphatemia at a specialized center in Buenos Aires, Argentina.

Archives of endocrinology and metabolism
2025

Significance of assessing the severity of craniosynostosis in patients with X-linked hypophosphatemia (XLH) at diagnosis.

Clinical pediatric endocrinology : case reports and clinical investigations : official journal of the Japanese Society for Pediatric Endocrinology
2025

Phenotypic characterization of ENPP1 deficiency: generalized arterial calcification of infancy and autosomal recessive hypophosphatemic rickets type 2.

JBMR plus
2025

Tumor-induced rickets/osteomalacia (TIO): diagnostic pitfalls and therapeutic options.

Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research
2025

Office Blood Pressure and Obesity in Children with X-Linked Hypophosphatemia.

Calcified tissue international
2025

Surgical treatment of the varus knee caused by vitamin D-resistant rickets. Report of two cases and review of the literature.

International journal of surgery case reports
2025

FGF23-Mediated Hypophosphatemic Rickets: Phenotype, Genotype, and Comparison to Non-FGF23-Mediated Forms.

Indian pediatrics
2025

Methodology for the international working group clinical practice guidelines on X-linked hypophosphatemia in children and adults.

Journal of bone and mineral metabolism
2025

Current Practices in Monitoring Children and Adults With X-linked Hypophosphatemia: A Global Survey of Expert Experience.

The Journal of clinical endocrinology and metabolism
2025

Prevalence of Comorbid Hyperparathyroidism and Its Association with Renal Dysfunction in Asian Patients with X-Linked Hypophosphatemic Rickets/Osteomalacia.

Calcified tissue international
2025

Disease burden and health-related quality of life among children with X-linked hypophosphataemia in China: a national cross-sectional survey.

BMJ paediatrics open
2025

X-linked hypophosphataemia.

Tidsskrift for den Norske laegeforening : tidsskrift for praktisk medicin, ny raekke
2025

X-Linked Hypophosphataemia and Burosumab: A Systemic Disease With a New Treatment.

Journal of paediatrics and child health
2025

Dental Manifestations in Children Affected by Hypophosphatemic Rickets: A Systematic Review and Meta-Analysis.

Children (Basel, Switzerland)
2025

Pain, Quality of Life, and Mental Health in Adults with X-linked Hypophosphatemia: A Cross-sectional Study.

The Journal of clinical endocrinology and metabolism
2025

X-Linked Hypophosphatemia Management in Children: An International Working Group Clinical Practice Guideline.

The Journal of clinical endocrinology and metabolism
2025

Burosumab Treatment in a Girl With Cutaneous Skeletal Hypophosphatemia Syndrome: 2-Year Follow-Up.

American journal of medical genetics. Part A
2025

Hypophosphatemic rickets in an Italian multicentric cohort of 24 subjects: a clinical and molecular characterisation.

Endocrine
2025

A deep intronic PHEX variant associated with X-linked hypophosphatemia in a Finnish family.

JBMR plus
2024

Hypophosphatemia in pregnancy: A case report.

Nigerian medical journal : journal of the Nigeria Medical Association
2025

18F-Sodium Fluoride PET/CT as a Tool to Assess Enthesopathies in X-Linked Hypophosphatemia.

Calcified tissue international
2025

Etiology, clinical characteristics, genetic profile, and outcomes of children with refractory rickets at a referral center in India: a cohort study.

Pediatric nephrology (Berlin, Germany)
2024

Hypophosphatemic Rickets as a Key Sign for the Diagnosis of Hereditary Tyrosinemia Type 1: Case Reports and Narrative Review of the Literature.

Revista medica de Chile
2024

Case report: Prolonged and severe hungry bone syndrome after parathyroidectomy in X-linked hypophosphatemia.

Frontiers in endocrinology
2025

Clinical practice recommendations for the diagnosis and management of X-linked hypophosphataemia.

Nature reviews. Nephrology
2025

Clinical, Radiographic, and Molecular Analysis of Patients with X-Linked Hypophosphatemic Rickets: Looking for Phenotype-Genotype Correlation.

Diagnostics (Basel, Switzerland)
2025

Systematic Review: Efficacy of Medical Therapy on Outcomes Important to Pediatric Patients With X-Linked Hypophosphatemia.

The Journal of clinical endocrinology and metabolism
2025

Respiratory insufficiency as a rare presentation in a child with vitamin D-dependent rickets type 1.

BMJ case reports
2025

Base of Skull & Spinal Canal Narrowing in an Adolescent with Autosomal Recessive Hypophosphatemic Rickets Type 2.

Calcified tissue international
2025

Mutant Fam20c knock-in mice recapitulate both lethal and non-lethal human Raine Syndrome.

BMC molecular and cell biology
2025

Vitamin D Dependent Rickets 2A With Alopecia: Three Cases With Novel Genetic Variants.

Pediatric dermatology
2025

Systematic Review: Efficacy of Medical Therapy on Outcomes Important to Adult Patients With X-Linked Hypophosphatemia.

The Journal of clinical endocrinology and metabolism
2025

Intragenic duplication of PHEX in a girl with X-linked hypophosphatemia: a case report with review of literature.

Endocrine journal
2024

Nephrocalcinosis tendency does not worsen under burosumab treatment for X-linked hypophosphatemic rickets: a multicenter pediatric study.

Frontiers in pediatrics
2025

Unusual PHEX variants implicate uncommon genetic mechanisms for X-linked hypophosphatemic rickets.

JBMR plus
2024

Phosphorus-independent role of FGF23 in erythropoiesis and iron homeostasis.

PloS one
2024

Use of Whole-Exome Sequencing and Pedigree Analysis to Identify X-linked Hypophosphatemia in Saudi Arabian Families.

Journal of the Endocrine Society
2025

No longer to be ignored: Hypophosphatemia following intravenous iron administration.

Reviews in endocrine &amp; metabolic disorders
2025

Newly identified intronic and known pathogenic point mutations in SLC34A3/NPT2c cause hereditary hypophosphatemic rickets with hypercalciuria.

Genes &amp; diseases
2024

The care of the patient with X-linked hypophosphatemic rickets.

European journal of paediatric dentistry
2024

Metabolic Bone Disease: An Overview.

Missouri medicine
2025

Genetic, Clinical, and Biochemical Characterization of a Large Cohort of Palestinian Patients With Fanconi-Bickel Syndrome.

Clinical genetics
2025

Bone Microarchitecture Evaluated by HR-PQCT in Chinese Adolescent and Pediatric Patients With X-Linked Hypophosphatemia.

The Journal of clinical endocrinology and metabolism
2025

Tertiary hyperparathyroidism in two paediatric patients with X-linked hypophosphatemia during Burosumab treatment.

Bone
2025

RNA-first Approach Identifies Deep Intronic PHEX Variants in X-linked Hypophosphatemic Rickets.

The Journal of clinical endocrinology and metabolism
2024

Burosumab, a Transformational Treatment in a Pediatric Patient With Cutaneous-Skeletal Hypophosphatemia Syndrome.

JCEM case reports
2024

Impact of X-linked hypophosphatemic rickets/osteomalacia on health and quality of life: baseline data from the SUNFLOWER longitudinal, observational cohort study.

JBMR plus
2025

Effective bone healing after corrective osteotomy in a patient with FGF23-related hypophosphatemic disease using short-term burosumab treatment.

Journal of the Formosan Medical Association = Taiwan yi zhi
2024

Lower limb maltorsion and acetabular deformity in children and adolescents with X-linked hypophosphatemia.

Frontiers in endocrinology
2025

Pharmacodynamic Exposure-Response Analysis of Fracture Count Data Following Treatment with Burosumab in Patients with XLH.

Journal of clinical pharmacology
2024

Clinical presentation and burden of ENPP1 deficiency in adults.

Archives de pediatrie : organe officiel de la Societe francaise de pediatrie
2024

Autosomal recessive hypophosphatemic rickets type 2 due to ENPP1 deficiency (ARHR2).

Archives de pediatrie : organe officiel de la Societe francaise de pediatrie
2024

ENPP1 deficiency: almost ready for prime time!

Archives de pediatrie : organe officiel de la Societe francaise de pediatrie
2024

FGF23 and Cell Stress in SaOS-2 Cells-A Model Reflecting X-Linked Hypophosphatemia Dynamics.

Cells
2024

[Clinical value of renal phosphorus threshold in the diagnosis and treatment X-linked hypophosphatemic rickets in children].

Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics
2024

Rare diseases: a challenge in paediatric dentistry.

European journal of paediatric dentistry
2024

Meta-analysis and systematic review: burosumab as a promising treatment for children with X-linked hypophosphatemia.

Frontiers in endocrinology
2024

Commentary on: The use of burosumab to treat autosomal-recessive hypophosphatemic rickets type 2: rationale and a first clinical experience.

Journal of nephrology
2024

X-linked hypophosphatemic rickets and nephrocalcinosis: clinical characteristics of a single-center pediatric cohort in North America before and after burosumab.

Frontiers in pediatrics
2024

Nephrocalcinosis and kidney function in children and adults with X-linked hypophosphatemia: baseline results from a large longitudinal study.

Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research
2025

Alteration of Bone Microarchitecture in Hereditary Distal RTA Patients With SLC4A1 Gene Mutation: Assessed by HR-pQCT.

The Journal of clinical endocrinology and metabolism
2024

Item Response Theory Quantifies the Relationship Between Improvements in Serum Phosphate and Patient-Reported Outcomes in Adults With X-Linked Hypophosphatemia.

Clinical pharmacology and therapeutics
2024

Inherited phosphate and pyrophosphate disorders: New insights and novel therapies changing the oral health landscape.

Journal of the American Dental Association (1939)
2024

Metabolically healthy obesity in adults with X-linked hypophosphatemia.

European journal of endocrinology
2024

X-linked hypophosphatemia: long-term outcomes of different treatment strategies.

Nefrologia
2024

Evaluation of dental manifestations in X-linked hypophosphatemia using orthopantomography.

PloS one
2024

A toddler with severe vitamin D-dependent rickets type 1 A (VDDR1A), hungry bone syndrome, and severe RSV infection: presentation and therapeutic challenges.

Hormones (Athens, Greece)
2024

Longitudinal assessment of physical function in adults with X-linked hypophosphatemia following initiation of burosumab therapy.

Osteoporosis international : a journal established as result of cooperation between the European Foundation for Osteoporosis and the National Osteoporosis Foundation of the USA
2024

Hereditary vitamin D resistant rickets (HVDRR) case series: phenotype, genotype, conventional treatment, and adjunctive cinacalcet therapy.

Pediatric endocrinology, diabetes, and metabolism
2024

NFATc1 Is Required for Vitamin D- and Phosphate-Mediated Regulation of Osteocyte Lacuno-Canalicular Remodeling.

Endocrinology
2024

Burosumab treatment of X-linked hypophosphatemia patients: interim analysis of the SUNFLOWER longitudinal, observational cohort study.

JBMR plus
2024

[Retrospective study on the diagnosis, treatment, and follow-up of 85 cases of hypophosphatemic rickets in children].

Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics
2024

Autosomal Recessive Hypophosphatemic Rickets Type 2 Associated with a Novel ENPP1 Variant in a Taiwanese Girl.

Journal of clinical research in pediatric endocrinology
2024

Lessons learned from the real-world diagnosis and management of hereditary hypophosphatemic rickets.

Bone reports
2024

Consenso mexicano de tirosinemia tipo 1.

Boletin medico del Hospital Infantil de Mexico
2024

Burosumab Efficacy and Safety in Patients with X-Linked Hypophosphatemia: Systematic Review and Meta-analysis of Real-World Data.

Calcified tissue international
2024

Coexistence of hypogonadotropic hypogonadism and hypophosphatemic rickets.

BMJ case reports
2024

Hypophosphatemic rickets and short stature.

Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research
2025

A de novo <italic>PRPF8</italic> Pathogenic Variant in Transient Severe Hypophosphatemia with Delayed Puberty and Growth Failure.

Hormone research in paediatrics
2024

Evaluation of bone density and microarchitecture in adult patients with X-linked hypophosphatemic rickets: A pilot longitudinal study.

Bone
2024

[Effect and safety of Burosumab in the treatment of 4 children with X-linked hypophosphatemia].

Zhonghua er ke za zhi = Chinese journal of pediatrics
2024

Safety and efficacy of burosumab in improving phosphate metabolism, bone health, and quality of life in adolescents with X-linked hypophosphatemic rickets.

European journal of medical genetics
2024

A Mild Presentation of X-Linked Hypophosphatemia Caused by a Non-Canonical Splice Site Variant in the PHEX Gene.

Genes
2024

Health-related quality of life of children with X-linked hypophosphatemia in Germany.

Pediatric nephrology (Berlin, Germany)
2024

Complex phenotype in Fanconi renotubular syndrome type 1: Hypophosphatemic rickets as the predominant presentation.

Clinica chimica acta; international journal of clinical chemistry
2024

Clinical characteristics and long-term management for patients with vitamin D-dependent rickets type II: a retrospective study at a single center in Saudi Arabia.

Frontiers in endocrinology
2025

Improved Oral Health in Adults With X-Linked Hypophosphatemia Treated With Burosumab.

The Journal of clinical endocrinology and metabolism
2024

CYP4A22 loss-of-function causes a new type of vitamin D-dependent rickets (VDDR1C).

Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research
2024

X-Linked hypophosphatemia. Data from a Spanish adult population cohort.

Journal of nephrology
2024

The role of GDF5 in regulating enthesopathy development in the Hyp mouse model of XLH.

Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research
2024

Siblings with vitamin D-dependent rickets type 1A: Importance of genetic testing and a review of genotype-phenotype correlations.

American journal of medical genetics. Part A
2024

Cardiovascular health in pediatric patients with X-linked hypophosphatemia under two years of burosumab therapy.

Frontiers in endocrinology
2024

Efficacy and safety of burosumab compared with conventional therapy in patients with X-linked hypophosphatemia: A systematic review.

Archives of endocrinology and metabolism
2024

Establishing a human-induced pluripotent stem cell line SMUSHi005-A from a patient with hypophosphatemic vitamin D-resistant rickets carrying the PHEX c.1586-1586+1 delAG mutation.

Stem cell research
2024

Identification of Rare and Novel PHEX Variants in X-linked Hypophosphatemia.

The Journal of clinical endocrinology and metabolism
2024

Recent advances in fibroblast growth factor 23-related hypophosphatemic disorders.

Current opinion in endocrinology, diabetes, and obesity
2024

Early manifestation of hypophosphatemic rickets in goslings: a potential role of insufficient muscular adenosine triphosphate in motility impairment of early P-deficient geese.

Poultry science
2024

Non-Surgical Strategies for Managing Skeletal Deformities in a Child with X-Linked Hereditary Hypophosphatemic Ricket: Insights and Perspectives.

Children (Basel, Switzerland)
2024

Hereditary hypophosphatemic rickets with hypercalciuria (HHRH), a complex disorder in need of precision medicine.

Kidney international
2024

Experience of X-linked hypophosphatemic rickets in the Gulf Cooperation Council countries: case series.

Endocrinology, diabetes &amp; metabolism case reports
Ver todos os 694 no EuropePMC

Associações

Organizações que acompanham esta doença — pra ter apoio e orientação

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Doença com base genética

Um médico geneticista pode ajudar no diagnóstico de Raquitismo hipofosfatêmico e no aconselhamento genético da família.

Vai consultar um especialista? Confirme o registro dele no conselho.
Conselhos e sociedades

Doenças relacionadas

Doenças com sintomas parecidos — ajudam quem ainda está buscando diagnóstico

Perguntas frequentes

O que as famílias mais perguntam sobre esta doença — cada resposta com a fonte de onde saiu

Respostas geradas por IA a partir das fontes citadas

É um grupo de doenças genéticas em que o corpo perde fosfato pela urina, gerando hipofosfatemia crônica com níveis normais de cálcio sérico. A carência de fósforo compromete a calcificação adequada dos ossos e a formação dos dentes.

Referências

Fontes citadas no texto, publicações do grafo e bases de dados usadas neste verbete

9 publicações do grafo RarasNet (PubMed) · 6 bases de dados. Títulos, periódicos e PMIDs vêm direto da fonte, sem intermediação de IA.

  1. ORPHA:437
    Orphanet
  2. GARD:6735
    GARD (NIH)
  3. Q1779987
    Wikidata

Dados compilados pelo RarasNet a partir de fontes abertas (Orphanet, OMIM, MONDO, PubMed/EuropePMC, ClinicalTrials.gov, DATASUS, PCDT/MS). Este conteúdo é informativo e não substitui avaliação médica.

Citar este verbete

Raras. (s.d.). Raquitismo hipofosfatêmico. Em Raras — Enciclopédia de Doenças Raras do Brasil. https://raras.org/doenca/raquitismo-hipofosfatemico

Formato APA. Conteúdo sob CC BY 4.0 — reuso livre com atribuição.

Conteúdo mantido por Agente Raras · Médicos e pesquisadores podem colaborar

Compêndio · Raras BR

Raquitismo hipofosfatêmico

ORPHA:437 · MONDO:0000044
🇧🇷 Brasil SUS
CEAF
1ABurosumabe
Geral
Prevalência
Unknown
Herança
Autosomal dominant, Autosomal recessive, X-linked dominant
CID-10
E83.31 · Distúrbios do metabolismo de minerais
CID-11
Ensaios
10 ativos
Início
All ages
Prevalência
0.0 (Worldwide)
MedGen
UMLS
C0733682
Repurposing
2 candidatos
dihydrotachysterol — vitamin analog
ergocalciferol
EuropePMC
Wikidata
Papers 10a
Evidência
🥇 Rev. sistemática

📋 Origem dos dados

Esta página agrega dados de fontes públicas e oficiais. Dados sobre cobertura no SUS (PCDT, CEAF) são verificados ativamente por agente proativo (ver badge no infobox). Demais dados têm atribuição de fonte + data da última sincronização — clique para abrir o original.

Doença rara (ontologia)
fonte: Orphanet
Identificador unificado
fonte: MONDO
Indexação biomédica
fonte: MeSH (NLM)
Dado público estruturado
fonte: Wikidata
Rara