Raras
Buscar doenças, sintomas, genes...
Síndrome McCune-Albright
ORPHA:562CID-10 · Q78.1CID-11 · FB80.0OMIM 174800DOENÇA RARA

A síndrome de McCune-Albright (SMA) é geralmente reconhecida pela presença de três características principais: displasia fibrosa dos ossos (DFO), manchas café com leite na pele e puberdade precoce (PP).

Mantido por Agente Raras·Colaborar como especialista →

Introdução

O que você precisa saber de cara

📋

A síndrome de McCune-Albright (SMA) é geralmente reconhecida pela presença de três características principais: displasia fibrosa dos ossos (DFO), manchas café com leite na pele e puberdade precoce (PP).

Pesquisas ativas
5 ensaios
24 total registrados no ClinicalTrials.gov
Publicações científicas
1.119 artigos
Último publicado: 2026 Apr 16
Medicamentos
3 registrados
PEGVISOMANT, FULVESTRANT, ANASTROZOLE

Tem tratamento?

3 medicamentos registrados
Ver detalhes, fases e interações →
PEGVISOMANTFULVESTRANTANASTROZOLE

Escala de raridade

CLASSIFICAÇÃO ORPHANET · BRASIL 2024
1-9 / 1 000 000
Ultra-rara
<1/50k
Muito rara
1/20k
Rara
1/10k
Pouco freq.
1/5k
Incomum
1/2k
Prevalência
0.55
Europe
Início
Childhood
🏥
SUS: Cobertura mínimaScore: 15%
CID-10: Q78.1
🇧🇷Dados SUS / DATASUS
PROCEDIMENTOS SIGTAP (5)
0202010503
Cariótipo — bandas G, Q ou Rgenetic_test
0202010600
Pesquisa de microdeleções/microduplicações por FISHlab_test
0202010694
Sequenciamento completo do exoma (WES)rehabilitation
0202010260
Dosagem de alfa-fetoproteína
0301070040
Atendimento em reabilitação — doenças raras
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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
14 sintomas
📏
Crescimento
9 sintomas
🫃
Digestivo
7 sintomas
😀
Face
4 sintomas
🫘
Rins
3 sintomas
👁️
Olhos
2 sintomas

+ 22 sintomas em outras categorias

Características mais comuns

100%prev.
Início juvenil
Obrigatório (100%)
100%prev.
Ílio esclerótico
Obrigatório (100%)
100%prev.
Dor óssea
Ocasional (29-5%)
100%prev.
Hipertireoidismo
Frequente (79-30%)
100%prev.
Displasia fibrosa poliostótica
Ocasional (29-5%)
100%prev.
Grandes máculas café-com-leite com margens irregulares
Muito frequente (99-80%)
66sintomas
Muito frequente (9)
Frequente (13)
Ocasional (21)
Muito raro (14)
Sem dados (9)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 66 características clínicas mais associadas, ordenadas por frequência.

Início juvenilJuvenile onset
Obrigatório (100%)100%
Ílio escleróticoSclerotic ilium
Obrigatório (100%)100%
Dor ósseaBone pain
Ocasional (29-5%)100%
HipertireoidismoHyperthyroidism
Frequente (79-30%)100%
Displasia fibrosa poliostóticaPolyostotic fibrous dysplasia
Ocasional (29-5%)100%

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa1desde 2026
Total histórico1.119PubMed
Últimos 10 anos200publicações
Pico202558 papers
Linha do tempo
2026Hoje · 2026🧪 1982Primeiro 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

1 gene identificado com associação a esta condição. Padrão de herança: Not applicable.

GNASProtein ALEXDisease-causing somatic mutation(s) inAltamente restrito
FUNÇÃO

May inhibit the adenylyl cyclase-stimulating activity of guanine nucleotide-binding protein G(s) subunit alpha which is produced from the same locus in a different open reading frame

LOCALIZAÇÃO

Cell membraneCell projection, ruffle

VIAS BIOLÓGICAS (10)
G alpha (s) signalling eventsProstacyclin signalling through prostacyclin receptorADORA2B mediated anti-inflammatory cytokines productionGPER1 signalingG alpha (i) signalling events
EXPRESSÃO TECIDUAL(Ubíquo)
Pituitária
1324.4 TPM
Tireoide
727.3 TPM
Hipotálamo
548.6 TPM
Brain Frontal Cortex BA9
501.2 TPM
Cérebro - Hemisfério cerebelar
474.1 TPM
OUTRAS DOENÇAS (12)
progressive osseous heteroplasiapituitary adenoma 3, multiple typespseudohypoparathyroidism type 1CMcCune-Albright syndrome
HGNC:4392UniProt:P84996

Medicamentos e terapias

PEGVISOMANTPhase 3

Mecanismo: Growth hormone receptor antagonist

FULVESTRANTPhase 2

Mecanismo: Estrogen receptor degrader

ANASTROZOLEPhase 2

Mecanismo: Cytochrome P450 19A1 inhibitor

Ver mais no OpenTargets

Variantes genéticas (ClinVar)

467 variantes patogênicas registradas no ClinVar.

🧬 GNAS: NM_016592.5(GNAS):c.138C>A (p.Ala46=) ()
🧬 GNAS: NM_016592.5(GNAS):c.195del (p.Asn66fs) ()
🧬 GNAS: NM_000516.7(GNAS):c.177G>C (p.Gln59His) ()
🧬 GNAS: NM_000516.7(GNAS):c.-2_1del (p.Met1del) ()
🧬 GNAS: NM_000516.7(GNAS):c.516del (p.Ile172fs) ()
Ver todas no ClinVar

Classificação de variantes (ClinVar)

Distribuição de 97 variantes classificadas pelo ClinVar.

19
78
Patogênica (19.6%)
VUS (80.4%)
VARIANTES MAIS SIGNIFICATIVAS
GNAS: NM_000516.7(GNAS):c.585+1G>C [Pathogenic]
GNAS: NM_000516.7(GNAS):c.433-2A>C [Pathogenic/Likely pathogenic]
GNAS: NM_000516.7(GNAS):c.970+1G>C [Pathogenic]
GNAS: NM_000516.7(GNAS):c.445_446del (p.His149fs) [Pathogenic/Likely pathogenic]
GNAS: NM_080425.4(GNAS):c.1263C>T (p.Asp421=) [Uncertain significance]

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.
Aprovado1
3Fase 32
2Fase 24
1Fase 11
·Pré-clínico9
Medicamentos catalogadosEnsaios clínicos· 3 medicamentos · 14 ensaios
Carregando informações de tratamento...

Onde tratar no SUS

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

🇧🇷 Atendimento SUS — Síndrome McCune-Albright

🗺️

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

🟢 Recrutando agora

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

Outros ensaios clínicos

24 ensaios clínicos encontrados, 5 ativos.

Distribuição por fase
Ver todos no ClinicalTrials.gov
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Publicações mais relevantes

Timeline de publicações
478 papers (10 anos)
#1

Addressing the unmet challenge of pain in rare bone diseases: new insights from the RUDY UK registry.

Orphanet journal of rare diseases2026 Jan 29

Pain is a common symptom in many rare bone disorders, often linked to depression and a substantial decline in quality of life. However, there is little information on the quality of the pain which may provide insights into pain mechanisms. This study aimed to describe and compare the frequency and characteristics of self-reported pain in adults with Fibrous Dysplasia of Bone/McCune-Albright Syndrome (FD/MAS), Osteogenesis Imperfecta (OI), and X-linked Hypophosphatemia (XLH). A cross-sectional study was conducted using the online UK RUDY registry. Adults with self -reported FD/MAS, OI, and XLH who completed the painDETECT questionnaire (PD-Q) were included. Pain prevalence and phenotypes were assessed using baseline PD-Q responses which were also mapped to a modified widespread pain index as a measure of generalized pain. Descriptive analyses were performed using R®. A total of 281 adults completed the baseline PD-Q (94 FD/MAS, 94 OI, and 93 XLH). Among these, 86% of patients currently experienced pain and 47% reported severe strongest pain in the past four weeks, with no significant differences between conditions. Pain prevalence and phenotype were similar across diseases, though pain sites differed. Neuropathic-like pain and female sex were significantly associated with poorer pain outcomes, including higher pain prevalence and intensity (p < 0.05). Generalized pain (18%) was significantly associated with moderate to severe anxiety (p = 0.03), depression (p < 0.001) and sleep impairment (p < 0.001). Despite distinct pathophysiological mechanisms, pain distribution appears similar across these bone diseases, suggesting a major role for non-skeletal factors. Generalized pain was frequent and associated with anxiety, depression, and sleep disturbances, suggesting nociplastic features maybe a significant driver of pain in adults with rare bone diseases. The online version contains supplementary material available at 10.1186/s13023-025-04167-4.

#2

A Case of Pediatric Adrenocortical Carcinoma With McCune-Albright Syndrome Masquerading as Spontaneously Regressing Neuroblastoma.

Pediatric blood &amp; cancer2026 Mar 15
#3

Genotype-phenotype correlation and challenges in mutation detection in McCune-Albright syndrome: a retrospective study of a French cohort.

Annales d'endocrinologie2026 Mar 19

McCune-Albright syndrome MAS is a rare mosaic disorder caused by post-zygotic GNAS activating mutations. MAS is characterized by fibrous dysplasia (FD) of the skeleton, café-au-lait skin macules, and hyperfunctioning endocrinopathies such as precocious puberty, thyroid disease, growth hormone excess, and FGF23-mediated phosphate wasting. Mosaicism leads to marked clinical heterogeneity and complicates molecular diagnosis. We retrospectively analyzed clinical and genotyping data of patients referred for suspected or clinically diagnosed MAS in a single French center (2014-2025). GNAS R201C and R201H mutations were detected by digital droplet PCR using peripheral blood as first-line samples, with additional testing of circulating cell-free, saliva, or tissue when indicated. We included 405 patients, from which 89 (22%) carried a GNAS mutation (52 R201C, 37 R201H). No significant clinical differences were observed between R201C and R201H. Among 578 analyzed samples, mutation detection varied by sample type, with the highest rates in tissue. Mutant allele frequency (MAF) in blood DNA was higher in patients with polyostotic than in FD (p = 0.0055), but was not associated with the overall MAS-related lesion number. No correlation was found between MAF and age at diagnosis. MAS shows substantial clinical and molecular heterogeneity without clear genotype-phenotype differences between R201 variants. Mutation detection strongly depends on sample type, reflecting disease mosaicism. A multimodal diagnostic strategy and larger collaborative cohorts are needed to optimize molecular diagnosis and refine genotype-phenotype correlations in MAS patients.

#4

Efficacy of Denosumab for Treatment of Pain in Fibrous Dysplasia and McCune-Albright Syndrome: A Systematic Review.

Clinical endocrinology2026 Mar 19

Fibrous dysplasia (FD) skeletal lesions have a range of clinical manifestations including pain, which is a major contributor to reduced quality of life in patients with FD/MAS. Denosumab has been shown to reduce skeletal lesion activity and improve radiographic bone density; however, there is mixed evidence regarding its effect on pain. The primary objective was to evaluate the efficacy of denosumab for the treatment of bone pain in FD/MAS. A systematic review was conducted according to PRISMA guidelines utilising electronic databases including PubMed, Cochrane Library, Embase, Web of Science, and ClinicalTrials.gov. Included studies were those that described patients with a diagnosis of FD, with or without MAS, who were treated with denosumab. The primary outcomes were change in pain score using quantitative pain scales (Visual Analogue Scale, Brief Pain Index, and patient-reported numerical rating scale), proportion of pain responders (≥ 2-point reduction in pain or an overall subjective improvement in pain), and change in analgesic requirement. The secondary outcome was adverse on-treatment and post-treatment events related to denosumab. There were 16 studies included (88 total patients, 86 treated with denosumab). Mean pain score decreased by 3.51 points on a 10-point scale. 81.8% of patients were pain responders, demonstrating a clinically meaningful improvement in pain. Reduction or cessation of analgesic use was reported in 51.7% of patients. Adverse events occurred in 55.8% of patients, most commonly hypocalcemia (11.6%) and hypophosphatemia (8.1%). Rebound hypercalcemia following denosumab discontinuation occurred in 8 patients (9.3%), 4 of which were severe. In patients with FD/MAS undergoing treatment with denosumab, pain improved in 81.8% of patients, and analgesic requirement decreased in greater than half of patients. Careful monitoring is required surrounding treatment due the potential for severe adverse events, particularly rebound hypercalcemia following treatment discontinuation.

#5

Precocious puberty: An overview of pathogenesis, clinical presentation, and management.

Best practice &amp; research. Clinical obstetrics &amp; gynaecology2026 Mar 04

Precocious puberty, defined as the onset of secondary sexual characteristics at 2.5 SD below the mean age of onset of puberty (8 years in girls and 9 years in boys) is a complex clinical condition that has considerable physiological and psychological consequences on children and their parents. There are two types - central (gonadotrophin-dependent) and peripheral (gonadotrophin independent). Among the causes of central precocious puberty are various genetic mutations, syndromes and central nervous system disorders that prematurely activate the hypothalamic pituitary gonadal axis leading to the secretion of sex steroids that induced pubertal changes. Peripheral precocious puberty is commonly secondary to isolated sources of sex steroids such as tumours, exogenous steroids or as part of the McCune Albright syndrome. Long-term consequences of precocious puberty especially the central type include short stature and psychological problems. Children presenting with features of precocious puberty (PP) must be thoroughly assessed starting with a detailed history, physical examination and initiation of appropriate investigations followed by categorisation of the PP. A multidisciplinary team (consisting of a paediatrician an adolescent gynaecologist, a paediatric endocrinologist, a geneticist and a clinical psychologist) is essential for management. Treatment should aim at arresting or reversing the pubertal changes, counselling and support both for the children and their families and, addressing the implications for genetic causes for the family (if any). Untreated, precocious puberty may have considerable negative psychological and medical impact on the child.

Publicações recentes

Ver todas no PubMed

📚 EuropePMC661 artigos no totalmostrando 195

2026

Genotype-phenotype correlation and challenges in mutation detection in McCune-Albright syndrome: a retrospective study of a French cohort.

Annales d'endocrinologie
2026

Efficacy of Denosumab for Treatment of Pain in Fibrous Dysplasia and McCune-Albright Syndrome: A Systematic Review.

Clinical endocrinology
2026

A Case of Pediatric Adrenocortical Carcinoma With McCune-Albright Syndrome Masquerading as Spontaneously Regressing Neuroblastoma.

Pediatric blood &amp; cancer
2026

Precocious puberty: An overview of pathogenesis, clinical presentation, and management.

Best practice &amp; research. Clinical obstetrics &amp; gynaecology
2026

cAMP/PKA signaling in endocrine hypertension: genetic mechanisms and pathophysiological insights.

Frontiers in endocrinology
2026

Comparison of traditional and new treatments for fibrous dysplasia: a systematic review and meta-analysis.

Osteoporosis international : a journal established as result of cooperation between the European Foundation for Osteoporosis and the National Osteoporosis Foundation of the USA
2026

Denosumab in pediatric patients with fibrous dysplasia/McCune-Albright syndrome: a single-center, open-labeled study.

Frontiers in endocrinology
2026

Anesthetic management of a patient with McCune-Albright syndrome complicated by pathological cervical fracture: a case report.

BMC anesthesiology
2026

Effects of anti-RANKL, Zoledronate or combination therapy in a mouse model of fibrous dysplasia: a preclinical study.

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

Medication-related osteonecrosis of the jaw in patient with McCune-Albright syndrome: A rare case report.

Journal of oral and maxillofacial pathology : JOMFP
2026

McCune-Albright Syndrome: A Case Series.

Cureus
2026

Optimizing Surgical Management of Craniofacial and Orbital Fibrous Dysplasia: A Multi-Center Retrospective Study.

Head &amp; neck
2026

Addressing the unmet challenge of pain in rare bone diseases: new insights from the RUDY UK registry.

Orphanet journal of rare diseases
2026

McCune-Albright Syndrome as a Rare Cause of Fanconi Syndrome and Kidney Failure: A Case Report and Literature Review.

Kidney medicine
2026

Burosumab treatment for FGF23-related hypophosphatemia in a two-year-old girl with McCune-Albright syndrome.

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

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

JBMR plus
2026

Delayed Diagnosis of McCune-Albright Syndrome in Adulthood Revealed by Spinal Fibrous Dysplasia: Report of 2 Cases.

JCEM case reports
2026

No Regression With Imatinib Treatment for Craniofacial Fibrous Dysplasia Associated With McCune-Albright Syndrome.

Journal of pediatric hematology/oncology
2025

The Natural Course of Pain in Fibrous Dysplasia/McCune Albright Syndrome: A Prospective Follow Up Study.

Calcified tissue international
2025

The GNAS Gene: Fibrous Dysplasia, McCune-Albright Syndrome, and Skeletal Structure and Function.

Genes
2026

Polyostotic fibrous dysplasia with neuro-axial involvement: a case report and clinical insights.

Acta neurologica Belgica
2026

Craniofacial fibrous dysplasia: Long-term postoperative outcomes in a retrospective case series with up to 40 years of follow-up.

Bone
2025

Cross-union surgery for tibiofibular fractures in McCune-Albright syndrome: a case report and literature review.

Frontiers in pediatrics
2025

Peripheral Precocious Puberty Due to Autonomous Gonadal Activation: A Multicenter Experience.

Cureus
2025

Growth and pubertal outcome of three-years medical treatment of peripheral precocious puberty in a boy with McCune-Albright Syndrome: a case report.

BMC pediatrics
2025

Clinical application of radiofrequency echographic multi-spectrometry (REMS) for diagnosis and follow-up in several rare bone disorders: a case series.

BMC medical imaging
2026

Childhood fibrous dysplasia.

Orthopaedics &amp; traumatology, surgery &amp; research : OTSR
2025

A rare case of McCune-Albright syndrome in a young male with hyperthyroidism and hypertrophic scars.

Oxford medical case reports
2025

Long-Term Follow-Up of a Patient with McCune-Albright Syndrome: A Case Report.

Journal of clinical medicine
2025

McCune-Albright syndrome with multiple hyperfunctional endocrinopathies: diagnosis, treatment, and long-term follow-up: a case report.

Frontiers in endocrinology
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

Plasma proteomic markers of pain and emotional dysfunction in fibrous dysplasia/McCune-Albright syndrome.

Bone
2025

Management of pediatric testicular microlithiasis.

Frontiers in pediatrics
2025

Endoscopic endonasal transsphenoidal approach of pituitary macroadenoma and optic canal stenosis in a patient with McCune-Albright syndrome.

Quantitative imaging in medicine and surgery
2025

Genotype-Phenotype Correlation in Fibrous Dysplasia/McCune-Albright Syndrome Patients With Craniofacial Lesions.

Oral diseases
2025

[Malignancy of fibrous dysplasia of the calvarial bone in patient with McCune-Albright syndrome: clinical observation and literature review].

Zhurnal voprosy neirokhirurgii imeni N. N. Burdenko
2025

Early Manifestation of McCune-Albright Syndrome in a 32-Month-Old Female: A Rare Case Report.

Journal of investigative medicine high impact case reports
2025

Single-cell analysis of human fibrous dysplasia bone reveals a fibrotic transcriptome and GNAS variants in endothelial, perivascular, and stromal cells.

American journal of human genetics
2025

Skeletal and endocrine manifestations of McCune-Albright syndrome in patients with fibrous dysplasia.

Journal of plastic, reconstructive &amp; aesthetic surgery : JPRAS
2025

Burosumab treatment for fibroblast growth factor-23-associated hypophosphatemia in an adult patient with severe fibrous dysplasia in McCune-Albright syndrome: case report and review of the literature.

JBMR plus
2025

Fibrous dysplasia/McCune-Albright syndrome: state-of-the-art advances, pathogenesis, and basic/translational research.

Orphanet journal of rare diseases
2025

Thyroid Scan Conundrum in a Rare Case of McCune-Albright Syndrome.

Indian journal of nuclear medicine : IJNM : the official journal of the Society of Nuclear Medicine, India
2025

[Precocious puberty in the McCune-Albright Syndrome: a case report].

The Pan African medical journal
2025

Segmental macular hyperpigmentation: new genes, new clinical implications.

The British journal of dermatology
2025

McCune-Albright syndrome: a case of an adult with fibrous dysplasia, severe cardiopulmonary complications, acromegaly, and chronic myeloid leukemia.

JBMR plus
2025

Update on the medical management of fibrous dysplasia of the bone.

Therapeutic advances in endocrinology and metabolism
2025

RASopathies. Part II: Cutaneous and extracutaneous manifestations.

Journal of the American Academy of Dermatology
2025

Surgical Management of Fibrous Dysplasia and Associated Syndromes.

The Journal of the American Academy of Orthopaedic Surgeons
2025

[Clinical characteristics and healthcare burden in patients with McCune-Albright syndrome].

Zhonghua er ke za zhi = Chinese journal of pediatrics
2025

RASopathies. Part I: Genetics and therapeutic considerations.

Journal of the American Academy of Dermatology
2025

[Malignant transformation of polyostotic fibrous dysplasia in long bone: a clinicopathological analysis of four cases].

Zhonghua bing li xue za zhi = Chinese journal of pathology
2025

Use of burosumab in McCune Albright syndrome: case report and review of literature in mosaic disorders with FGF23 overproduction.

Frontiers in endocrinology
2025

A Splice-Region Variant Causes an Atypical Presentation of GNAS Inactivation Disorder.

American journal of medical genetics. Part A
2025

Peripheral precocious puberty in girls with McCune-Albright syndrome: a case series.

Archives of endocrinology and metabolism
2025

Safe Continuation of Pegvisomant During Pregnancy in a Patient With Fibrous Dysplasia/McCune-Albright Syndrome.

JCEM case reports
2025

Case Report: Late diagnosis of McCune-Albright with severe kyphoscoliosis, acromegaly and tertiary hyperparathyroidism.

Frontiers in endocrinology
2025

A Dominant Mutation in Gαs-Protein Increases Hair Pigmentation.

Pigment cell &amp; melanoma research
2025

Intramuscular Myxoma in the Vastus Medialis Muscle: A Case Report and Brief Review of the Literature.

Journal of orthopaedic case reports
2025

Bone pain in fibrous dysplasia does not rely on aberrant sensory nerve sprouting or neuroma formation.

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

Developing a Standardised Dataset for Natural History Studies in Fibrous Dysplasia/McCune-Albright Syndrome.

Calcified tissue international
2025

Burosumab treatment of a child with McCune-Albright syndrome/polyostotic fibrous dysplasia: challenges and benefits.

JBMR plus
2025

Nonmalignant Adrenocorticotrophic Hormone-Independent Cushing's Syndrome in Pediatric Patients: A Retrospective Observational Cohort Study.

Hormone research in paediatrics
2025

Clinical spectrum and uncommon features of McCune-Albright syndrome in children: a cohort study from a National Referral Center.

Frontiers in endocrinology
2025

Analysis of Series of Cases of Fibrous Dysplasia of Proximal Femur in Pediatric Population.

Journal of orthopaedic case reports
2025

Two-stage surgical strategy for extensive craniofacial fibrous dysplasia with cerebral compression.

Acta neurochirurgica
2025

A Case of McCune-Albright Syndrome with External Auditory Canal Stenosis Treated with Image-Guided Surgery System-Assisted Temporal Bone Surgery.

The journal of international advanced otology
2025

A Case of McCune Albright Syndrome With Rare Extensive Polyostotic Fibrous Dysplasia.

Clinical nuclear medicine
2024

Posterior Reversible Encephalopathy Syndrome in the Context of McCune-Albright Syndrome: A Case Report.

Cureus
2025

Syndromic fibrous dysplasia of the proximal femur.

BMJ case reports
2024

Fertility journey of a patient with McCune-Albright syndrome associated with bilateral ovarian involvement.

F&amp;S reports
2025

A Qualitative Approach to Quality of Life in Fibrous Bone Dysplasia /McCune Albright Syndrome: Looking Beyond Quantitative Analysis.

Calcified tissue international
2025

Identifying Pain Subtypes in Patients With Craniofacial Lesions of Fibrous Dysplasia/McCune-Albright Syndrome.

Journal of oral and maxillofacial surgery : official journal of the American Association of Oral and Maxillofacial Surgeons
2024

Synchronous bilateral adrenalectomy for ACTH-independent Cushing's syndrome in children: multidisciplinary management.

European journal of endocrinology
2025

Safety and Efficacy of Moderate-dose Denosumab in Fibrous Dysplasia: Observational Results From a Phase 2 Clinical Trial.

The Journal of clinical endocrinology and metabolism
2024

McCune-Albright Syndrome: A Case From Mauritius.

Cureus
2024

Pituitary Acrogigantism: From the Past to the Future.

Frontiers of hormone research
2025

Rigid intramedullary nailing of lower limb segments in children and adolescents with metabolic bone disease.

Journal of pediatric orthopedics. Part B
2024

McCune-Albright Syndrome.

The New England journal of medicine
2024

"At the end of the tunnel": Endonasal endoscopy for a giant GH secreting pituitary tumor with conchal dysplastic sphenoid in McCune-Albright syndrome.

Journal of clinical neuroscience : official journal of the Neurosurgical Society of Australasia
2024

Diagnosis and management of pituitary adenomas in children and adolescents.

European journal of endocrinology
2025

Malignant transformation of craniofacial fibrous dysplasia: A clinicopathological, immunohistochemical and molecular analysis of 15 cases in one single institution.

Journal of stomatology, oral and maxillofacial surgery
2024

Exploring mutations: GNAS and CDC73 in jaw fibroosseous lesions.

Pathology, research and practice
2024

Improvement of Fibrous Dysplasia After Burosumab Therapy in a Pediatric Patient with McCune-Albright Syndrome: A Case Report.

JBJS case connector
2024

Dissecting the heterogeneity of craniofacial lesions in patients with fibrous dysplasia/McCune-Albright Syndrome.

International journal of oral and maxillofacial surgery
2024

[Analysis of clinical features of 193 Chinese patients with McCune-Albright syndrome through a literature review].

Zhonghua yi xue yi chuan xue za zhi = Zhonghua yixue yichuanxue zazhi = Chinese journal of medical genetics
2024

Fibrous dysplasia: A tale of two syndromes.

SA journal of radiology
2024

Craniomaxillofacial Fibro-osseous Lesions in Children.

Oral and maxillofacial surgery clinics of North America
2024

Presentation and Care for Children with Peripheral Precocious Puberty.

Endocrinology and metabolism clinics of North America
2025

Diagnostic Conundrum of a Sertoli Cell Tumor in a 2-Year-Old Girl with Peripheral Precocious Puberty and a Café-au-Lait Macule: A Case Report.

Hormone research in paediatrics
2024

Fibrocartilaginous Dysplasia of the Proximal Femur in Two Pediatric Patients, Including a Pathologic Fracture in a Patient With McCune-Albright Syndrome.

Fetal and pediatric pathology
2024

Heterozygous gain of function variant in GUCY1A2 may cause autonomous ovarian hyperfunction.

European journal of endocrinology
2024

Recent research advances in pain mechanisms in McCune-Albright syndrome thinking about the pain mechanism of FD/MAS.

Journal of orthopaedic surgery and research
2024

RNA-based bone histomorphometry: method and its application to explaining postpubertal bone gain in a G610C mouse model of osteogenesis imperfecta.

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

Acquired Forms of Fibroblast Growth Factor 23-Related Hypophosphatemic Osteomalacia.

Endocrinology and metabolism (Seoul, Korea)
2024

McCune-Albright syndrome: Beyond classical craniofacial deformities.

Joint bone spine
2024

[Research progress on the pathogenic mechanisms, diagnosis and treatment of McCune-Albright syndrome].

Zhonghua yu fang yi xue za zhi [Chinese journal of preventive medicine]
2024

A young woman with atypical McCune-Albright syndrome and the difficult road to recovery: a case report.

Frontiers in surgery
2024

A pathogenic role for brain-derived neurotrophic factor (BDNF) in fibrous dysplasia of bone.

Bone
2024

Diagnostic journey for individuals with fibrous dysplasia / McCune albright syndrome (FD/MAS).

Orphanet journal of rare diseases
2024

ERN BOND: The key European network leveraging diagnosis, research, and treatment for rare bone conditions.

European journal of medical genetics
2024

[Expert consensus on diagnosis and management of McCune-Albright syndrome in children (2023)].

Zhonghua er ke za zhi = Chinese journal of pediatrics
2024

Choosing the Best Tissue and Technique to Detect Mosaicism in Fibrous Dysplasia/McCune-Albright Syndrome (FD/MAS).

Genes
2023

Fibrous Dysplasia Masquerading as Sternal Malignancy: A Rare and Challenging Presentation.

Cureus
2023

Adrenal Cushing's syndrome in children.

Frontiers in endocrinology
2024

Incidence and Prevalence of Fibrous Dysplasia/McCune-Albright Syndrome: A Nationwide Registry-Based Study in Denmark.

The Journal of clinical endocrinology and metabolism
2023

[McCune-Albright syndrome: a case report and literature review].

The Pan African medical journal
2024

Pasireotide: potential treatment option for McCune-Albright-associated acromegaly.

European journal of endocrinology
2023

Fibula allograft transplantation combined with locking plate for treatment of recurrent monostotic fibular fibrous dysplasia: A case report.

World journal of clinical cases
2023

The Facial Osteoplasty for Polyostotic Fibrous Dysplasia in a Patient With McCune-Albright Syndrome: A Case Report.

Cureus
2025

Formulation and characterisation of metyrapone suppositories for the first effective long-term use in an infant with McCune-Albright syndrome-related Cushing syndrome.

European journal of hospital pharmacy : science and practice
2024

Inherited fibroblast growth factor 23 excess.

Best practice &amp; research. Clinical endocrinology &amp; metabolism
2023

Precocious Puberty: Types, Pathogenesis and Updated Management.

Cureus
2025

Successful Use of Metyrapone Suppositories in an Infant with Neonatal Cushing and McCune Albright Syndrome: A Case Report.

Hormone research in paediatrics
2024

McCune-Albright syndrome: Anesthetic management of cesarean section.

International journal of obstetric anesthesia
2023

The 'Eye Mask Sign' in Fibrous Dysplasia on 99mTc-MDP Skeletal Scintigraphy and SPECT/CT.

Nuclear medicine and molecular imaging
2024

Serum Phosphorus as a Driver of Skeletal Morbidity in Fibrous Dysplasia.

The Journal of clinical endocrinology and metabolism
2023

Diagnosis and treatment of McCune-Albright syndrome: A case report.

World journal of clinical cases
2023

Neonatal cholestasis as the onset symptom of McCune-Albright syndrome: case reports and a literature review.

Frontiers in pediatrics
2023

[The enigma of Henry IV's disease: Did he suffer from McCune-Albright syndrome/fibrous dysplasia?].

Revista espanola de patologia : publicacion oficial de la Sociedad Espanola de Anatomia Patologica y de la Sociedad Espanola de Citologia
2023

A Rare Case of Headache in a Patient With McCune-Albright Syndrome: A Triple Threat.

Cureus
2024

RANK-L inhibitor as a promising agent for refractory extensive craniofacial fibrous dysplasia: A case report.

Head &amp; neck
2024

Phenotyping Pain in Patients With Fibrous Dysplasia/McCune-Albright Syndrome.

The Journal of clinical endocrinology and metabolism
2023

Efficacy of antiresorptive agents in fibrous dysplasia and McCune Albright syndrome, a systematic review and meta-analysis.

Reviews in endocrine &amp; metabolic disorders
2023

GH-secreting pituitary adenoma in the course of McCune‑Albright syndrome in a 21-year-old patient complicated by hepatocellular carcinoma.

Endokrynologia Polska
2023

Case Report: Severe McCune-Albright syndrome presenting with neonatal Cushing syndrome: navigating through clinical obstacles.

Frontiers in endocrinology
2023

The longest reported survival of a child with McCune-Albright syndrome and a severe early presenting phenotype consisting of neonatal cushing syndrome, cardiac and liver diseases.

Journal of surgical case reports
2023

Lesion Expansion in Gnathic Fibrous Dysplasia: Natural History, Indicators of Progression, and Response to Bisphosphonates.

Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research
2023

Brain and/or Spinal Cord Tumors Accompanied with Other Diseases or Syndromes.

Advances in experimental medicine and biology
2023

Lower-limb intramedullary nailing in patients with polyostotic fibrous dysplasia who had a previous unsuccessful treatment. A report of 48 cases.

Journal of orthopaedics and traumatology : official journal of the Italian Society of Orthopaedics and Traumatology
2024

Clinical course of peripheral precocious puberty in girls due to autonomous ovarian cysts.

Clinical endocrinology
2022

[Clinical features of Chilean patients with Fibrous Dysplasia/McCune-Albright Syndrome].

Revista medica de Chile
2023

[Further progress of the etiology,diagnosis and treatment of peripheral precocious puberty].

Zhonghua yu fang yi xue za zhi [Chinese journal of preventive medicine]
2023

Severe osteoporosis in a young man with bilateral Cushing's syndrome: a case report.

Journal of medical case reports
2023

Denosumab use in bone fibrous dysplasia refractory to bisphosphonate: A retrospective multicentric study.

Bone
2023

Successful ART outcome in a woman with McCune-Albright syndrome: a case report and literature review.

Journal of assisted reproduction and genetics
2023

Brain and eye involvement in McCune-Albright Syndrome: clinical and translational insights.

Frontiers in endocrinology
2023

Normal Adult Height in an Untreated Boy With McCune-Albright Syndrome Presenting With Precocious Puberty.

AACE clinical case reports
2023

McCune-Albright Syndrome: A Case Report and Review of Literature.

International journal of molecular sciences
2023

Mazabraud Syndrome Associated with McCune-Albright Syndrome: A Case Report and Literature Review.

Case reports in oncology
2023

Pituitary Tumorigenesis-Implications for Management.

Medicina (Kaunas, Lithuania)
2023

Coxa Vara Deformity in Fibrous Dysplasia/McCune-Albright Syndrome: Prevalence, Natural History and Risk Factors: A Two-Center Study.

Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research
2023

Patient with McCune albright syndrome: Case report and 10 Years of follow-up imaging examination.

Journal of clinical and experimental dentistry
2023

Clinical value of RANKL, OPG, IL-6 and sclerostin as biomarkers for fibrous dysplasia/McCune-Albright syndrome.

Bone
2023

Pituitary tumours: molecular and genetic aspects.

The Journal of endocrinology
2023

Autonomous growth hormone secretion due to McCune Albright syndrome in paediatric age group: an ominous triad.

Endocrine
2023

Craniofacial Fibrous Dysplasia: Clinical and Therapeutic Implications.

Current osteoporosis reports
2023

Medication-Related Osteonecrosis of the Jaws (MRONJ) in Children and Young Patients-A Systematic Review.

Journal of clinical medicine
2023

Structural and Functional Implication of Natural Variants of Gαs.

International journal of molecular sciences
2023

Multiple subepidermal calcified nodule confined to café-au-lait spots.

The Journal of dermatology
2023

Low rates of neurological abnormalities in patients with pigmentary mosaicism: A retrospective cohort study from a tertiary dermatology center.

Pediatric dermatology
2023

Pharmacological Interventions Targeting Pain in Fibrous Dysplasia/McCune-Albright Syndrome.

International journal of molecular sciences
2023

[Identification of pathogenic variants in three Chinese patients with McCune-Albright syndrome].

Zhonghua yi xue yi chuan xue za zhi = Zhonghua yixue yichuanxue zazhi = Chinese journal of medical genetics
2022

Case report: Surgical treatment of McCune-Albright syndrome with hyperthyroidism and retrosternal goiter: A case report and literature review.

Frontiers in surgery
2023

Expression of RANKL in breast cancer tissue in patients with fibrous dysplasia/McCune-Albright syndrome.

Bone
2022

McCune-Albright syndrome with acromegaly: A case report with characteristic radiographic features of fibrous dysplasia.

Imaging science in dentistry
2023

Identification of GNAS Variants in Circulating Cell-Free DNA from Patients with Fibrous Dysplasia/McCune Albright Syndrome.

Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research
2022

Surgical correction of valgus deformities of the knee in Polyostotic Fibrous Dysplasia.

Orthopedic reviews
2022

A multidisciplinary care pathway improves quality of life and reduces pain in patients with fibrous dysplasia/McCune-Albright syndrome: a multicenter prospective observational study.

Orphanet journal of rare diseases
2023

Hyperthyroidism in McCune-Albright Syndrome - a case report.

Journal of pediatric endocrinology &amp; metabolism : JPEM
2022

[OPTIC DISC EDEMA IN McCUNE-ALBRIGHT SYNDROME].

Harefuah
2023

Burosumab Therapy in a Paediatric Patient with McCune-Albright Syndrome: A Case Report.

Hormone research in paediatrics
2022

Otalgia revealing McCune-Albright syndrome: A case report.

Annals of medicine and surgery (2012)
2024

McCune-Albright Syndrome: Vision Loss and Strabismus as the Initial Symptoms in a Child.

Journal of neuro-ophthalmology : the official journal of the North American Neuro-Ophthalmology Society
2022

Nonspecific Cystic Degeneration in Craniofacial Fibrous Dysplasia: A Rare Finding.

Contemporary clinical dentistry
2023

Phosphatonins: From Discovery to Therapeutics.

Endocrine practice : official journal of the American College of Endocrinology and the American Association of Clinical Endocrinologists
2023

Craniofacial fibrous dysplasia and aneurismal bone cyst in a patient with McCune-Albright syndrome. A case report and review of the literature.

Neurocirugia
2022

Large café-au-lait spots on a 5-year-old boy.

JAAD case reports
2022

Cushing syndrome as a failed cardiac screen in a patient with McCune-Albright syndrome: a case report.

Journal of medical case reports
2022

Malignant Sarcomatous Degeneration of Craniofacial Fibrous Dysplasia.

The Journal of craniofacial surgery
2022

McCune-Albright Syndrome in Infant with Growth Hormone Excess.

Genes
2022

Pathological Femoral Shaft Fracture With McCune-Albright Syndrome With Hyperthyroidism Managed With Oral Alendronate: A Case Report.

Cureus
2022

ACTH-independent Cushing's syndrome due to ectopic endocrinologically functional adrenal tissue caused by a GNAS heterozygous mutation: a rare case of McCune-Albright syndrome accompanied by central amenorrhea and hypothyroidism: a case report and literature review.

Frontiers in endocrinology
2022

Sleep Problems in Children With Neurocutaneous Syndromes: A Cross-Sectional Study.

Journal of child neurology
2022

68Ga-DOTA-TATE uptake in polyostotic fibrous dysplasia with McCune-Albright syndrome.

Revista espanola de medicina nuclear e imagen molecular
2022

A case report of McCune-Albright syndrome with hepatic manifestations.

Clinical case reports
2022

Surgical management of syndromic versus non-syndromic craniofacial fibrous dysplasia: a systematic review and meta-analysis.

The British journal of oral &amp; maxillofacial surgery
2022

Long-term imaging course of Chiari malformation type I due to fibrous dysplasia/McCune-Albright syndrome.

Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery
2023

Fibrous dysplasia and McCune-Albright syndrome: A case report with review of literature on the rehabilitation approach.

Turkish journal of physical medicine and rehabilitation
2022

Risk of developing spontaneous MRONJ in fibrous dysplasia patients treated with bisphosphonates: a systematic review of the literature.

Quintessence international (Berlin, Germany : 1985)
2022

Genetic Alterations in Benign Adrenal Tumors.

Biomedicines
2022

Effects of zoledronic acid therapy in fibrous dysplasia of bone: a single-center experience.

Archives of endocrinology and metabolism
2025

Management Strategies of Fibrous Dysplasia Involving the Paranasal Sinus and the Adjacent Skull Base.

Ear, nose, &amp; throat journal
2022

Case Report: A Neuro-Ophthalmological Assessment of Vision Loss in a Pediatric Case of McCune-Albright Syndrome.

Frontiers in medicine
2022

Molecular genetic testing in the management of pituitary disease.

Clinical endocrinology
2022

Surgical treatment of femoral deformities in polyostotic fibrous dysplasia and McCune-Albright syndrome: A literature review.

World journal of orthopedics
2022

Incidental Intercostal Intramuscular Myxoma With Fibrous Dysplasia in a Patient With Mazabraud's and McCune Albright Syndromes.

Cureus
2022

Neuropathic-like Pain in Fibrous Dysplasia/McCune-Albright Syndrome.

The Journal of clinical endocrinology and metabolism
2022

Postoperative thyroid crisis in an 11-year old male with McCune-Albright syndrome and atypical triiodothyronine hyperthyroidism: A case report.

Medicine
2022

Intramedullary Nailing for Lower Limb Polyostotic Fibrous Dysplasia in Children: A Long-term Follow-up Study.

Journal of pediatric orthopedics
2022

[Clinicopathological analysis of 105 patients with fibrous dysplasia of cranio-maxillofacial region].

Beijing da xue xue bao. Yi xue ban = Journal of Peking University. Health sciences
2022

A rare pediatric case of McCune-Albright syndrome with acute visual disturbance: Case report.

Medicine
2022

Inhibition of IL-6 in the treatment of fibrous dysplasia of bone: The randomized double-blind placebo-controlled TOCIDYS trial.

Bone
2021

Cutaneous mosaicism: Special considerations for women.

International journal of women's dermatology
2022

Clinicopathological and genetic study of a rare occurrence: Malignant transformation of fibrous dysplasia of the jaws.

Molecular genetics &amp; genomic medicine
2022

Treatment of fibrous dysplasia: focus on denosumab.

Expert opinion on biological therapy
Ver todos os 661 no EuropePMC

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Doenças relacionadas

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Referências e fontes

Bases de dados externas citadas neste artigo

Publicações científicas

Artigos indexados no PubMed ligados a esta doença no grafo RarasNet — título, periódico e PMID direto da fonte, sem intermediação de IA.

  1. Addressing the unmet challenge of pain in rare bone diseases: new insights from the RUDY UK registry.
    Orphanet journal of rare diseases· 2026· PMID 41612382mais citado
  2. A Case of Pediatric Adrenocortical Carcinoma With McCune-Albright Syndrome Masquerading as Spontaneously Regressing Neuroblastoma.
    Pediatric blood &amp; cancer· 2026· PMID 41834309mais citado
  3. Genotype-phenotype correlation and challenges in mutation detection in McCune-Albright syndrome: a retrospective study of a French cohort.
    Annales d'endocrinologie· 2026· PMID 41864326mais citado
  4. Efficacy of Denosumab for Treatment of Pain in Fibrous Dysplasia and McCune-Albright Syndrome: A Systematic Review.
    Clinical endocrinology· 2026· PMID 41858142mais citado
  5. Precocious puberty: An overview of pathogenesis, clinical presentation, and management.
    Best practice &amp; research. Clinical obstetrics &amp; gynaecology· 2026· PMID 41832867mais citado
  6. [Clinical analysis of 4 cases of McCune-Albright syndrome presenting with infantile cholestasis].
    Zhonghua Er Ke Za Zhi· 2026· PMID 41986270recente
  7. Genetics of Familial Acromegaly and Pituitary Gigantism.
    J Clin Endocrinol Metab· 2026· PMID 41965096recente
  8. The use of denosumab in rare bone diseases in adults: a systematic review from the ECTS Rare Bone Disease Action Group.
    J Clin Endocrinol Metab· 2026· PMID 41955566recente
  9. Letrozole effectively treats peripheral precocious puberty in a girl with McCune-Albright syndrome: a case report and review of the literature.
    BMC Pediatr· 2026· PMID 41947100recente
  10. Fibrous dysplasia in a patient with Carney complex.
    Hormones (Athens)· 2026· PMID 41926071recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:562(Orphanet)
  2. OMIM OMIM:174800(OMIM)
  3. MONDO:0018919(MONDO)
  4. GARD:6995(GARD (NIH))
  5. Variantes catalogadas(ClinVar)
  6. Busca completa no PubMed(PubMed)
  7. Artigo Wikipedia(Wikipedia)
  8. Q727008(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.

Conteúdo mantido por Agente Raras · Médicos e pesquisadores podem colaborar

Síndrome McCune-Albright
Compêndio · Raras BR

Síndrome McCune-Albright

ORPHA:562 · MONDO:0018919
Prevalência
1-9 / 1 000 000
Herança
Not applicable
CID-10
Q78.1 · Displasia poliostótica fibrosa
CID-11
Ensaios
5 ativos
Medicamentos
3 registrados
Início
Childhood
Prevalência
0.55 (Europe)
MedGen
UMLS
C0242292
EuropePMC
Wikidata
Wikipedia
Papers 10a
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