Uma condição que afeta a pele, o cérebro e os nervos, causada por um erro genético no processamento de gorduras pelo corpo. É caracterizada por pele seca, grossa e escamosa desde o nascimento, dificuldade de aprendizado e rigidez muscular.
Introdução
O que você precisa saber de cara
A síndrome de Sjögren-Larsson é uma condição que afeta a pele e os olhos, bem como o cérebro e a medula espinhal (sistema nervoso central).
Recém-nascidos com síndrome de Sjögren-Larsson tendem a nascer prematuramente. No nascimento, a pele pode estar mais espessa que o normal (hiperqueratose) e vermelha (eritema), mas geralmente torna‑se seca e escamosa (ichiose). Coceira de leve a grave (prurido) também é comum. As anomalias cutâneas geralmente afetam o pescoço, o tronco, os braços e as pernas. O rosto costuma não ser afetado.
Pequenos cristais no tecido sensível à luz na parte posterior do olho (retina) podem ser vistos durante um exame oftalmológico na maioria dos indivíduos afetados que têm mais de 3 a 4 anos de idade. Essa característica é única em pessoas com síndrome de Sjögren-Larsson e pode ser usada para diagnosticar a condição. Esses cristais retinianos são frequentemente chamados de “pontos brancos cintilantes”. Pessoas com síndrome de Sjögren-Larsson também podem apresentar miopia (visão curta) ou sensibilidade aumentada à luz (fotofobia).
Pessoas com síndrome de Sjögren-Larsson podem apresentar sinais e sintomas neurológicos. Indivíduos afetados frequentemente têm leucoencefalopatia, que é uma alteração em um tipo de tecido cerebral chamado substância branca. A substância branca é composta por fibras nervosas que são cobertas por uma substância (mielina) que isola e protege os nervos. A leucoencefalopatia acredita‑se que contribua para muitos dos sinais e sintomas neurológicos observados em pessoas com síndrome de Sjögren-Larsson.
A maioria das pessoas com síndrome de Sjögren-Larsson tem deficiências intelectuais, que podem variar de leves a graves. Dificuldades de fala (disartria) são comuns, e o desenvolvimento da fala costuma ser atrasado. Indivíduos afetados tipicamente têm mais dificuldade em falar do que em compreender a linguagem. Aproximadamente 35 a 40 por cento das pessoas com síndrome de Sjögren-Larsson têm convulsões.
Crianças com síndrome de Sjögren-Larsson tipicamente apresentam rigidez muscular anormal (espasticidade) nas pernas e, com menor frequência, nos braços. A espasticidade pode causar atraso no desenvolvimento de habilidades motoras como sentar, engatinhar e andar. Deformidades articulares (contraturas) também são comuns. Aproximadamente 50 por cento das pessoas com síndrome de Sjögren-Larsson precisarão de auxílio de cadeira de rodas.
Fonte: MedlinePlus Genetics (NLM/NIH), traduzidoUma condição que afeta a pele, o cérebro e os nervos, causada por um erro genético no processamento de gorduras pelo corpo. É caracterizada por pele seca, grossa e escamosa desde o nascimento, dificuldade de aprendizado e rigidez muscular.
Escala de raridade
<1/50kMuito rara
1/20kRara
1/10kPouco freq.
1/5kIncomum
1/2k
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Sinais e sintomas
O que aparece no corpo e com que frequência cada sintoma acontece
Partes do corpo afetadas
+ 11 sintomas em outras categorias
Características mais comuns
Os sintomas variam de pessoa para pessoa. Abaixo estão as 47 características clínicas mais associadas, ordenadas por frequência.
Linha do tempo da pesquisa
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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: Autosomal recessive.
Curadoria gene-doença
fontes oficiaisSjoegren-Larsson syndrome
An autosomal recessive neurocutaneous disorder characterized by a combination of severe intellectual disability, spastic di- or tetraplegia and congenital ichthyosis. Ichthyosis is usually evident at birth with varying degrees of erythema and scaling, neurologic symptoms appear in the first or second year of life. Most patients have an IQ of less than 60. Additional clinical features include glistening white spots on the retina, seizures, short stature and speech defects.
Variantes genéticas (ClinVar)
301 variantes patogênicas registradas no ClinVar.
Classificação de variantes (ClinVar)
Distribuição de 255 variantes classificadas pelo ClinVar.
Vias biológicas (Reactome)
6 vias biológicas associadas aos genes desta condição.
Diagnóstico
Os sinais que médicos procuram e os exames que confirmam
Tratamento e manejo
Remédios, cuidados de apoio e o que precisa acompanhar
Onde tratar no SUS
Hospitais de referência no Brasil e o protocolo oficial do SUS (PCDT)
🇧🇷 Atendimento SUS — Síndrome Sjögren-Larsson
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Pesquisa ativa
Ensaios clínicos abertos e novidades científicas recentes
Ensaios em destaque
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Pesquisa e ensaios clínicos
3 ensaios clínicos encontrados.
Publicações mais relevantes
Multisystem manifestations of Sjögren-Larsson syndrome in early childhood and its dental implications.
Sjögren-Larsson syndrome (SLS) constitutes a rare genetic disorder manifesting as a complex neurocutaneous condition characterised by congenital ichthyosis, progressive neurological impairment and intellectual disability. This case report presents an early childhood female patient exhibiting the classic triad of symptoms, along with significant oral complications, including severe dental caries, enamel demineralisation and gingivitis. Molecular genetic testing confirmed a homozygous pathogenic variant in the ALDH3A2 (Aldehyde Dehydrogenase 3 family member 2) gene, establishing the diagnosis. The patient's management encompassed a comprehensive multidisciplinary approach, integrating dental interventions under general anaesthesia, systemic therapies for spasticity and cutaneous manifestations, and regular follow-up care. This case highlights the critical importance of recognising oral manifestations in SLS and emphasises the need for integrated oral healthcare within the broader therapeutic framework for affected individuals.
General Anesthesia for a Child With Sjögren-Larsson Syndrome.
This case report describes the dental treatment performed under general anesthesia for a boy with Sjögren-Larsson syndrome (SLS), a rare autosomal recessive genetic disorder. The main symptoms of SLS are congenital ichthyosis, limb paraplegia or tetraplegia, and intellectual disability. The main concerns regarding the administration of general anesthesia to patients with SLS include difficulty with adhesion of the electrocardiogram electrodes and intravenous (IV) catheter dressings due to ichthyosis and heat intolerance secondary to hypohidrosis. Although it was first described approximately 70 years ago, there is little in the existing literature on general anesthesia for patients with SLS. We administered deep sedation/general anesthesia using continuous IV infusions of propofol and remifentanil along with a laryngeal mask airway in a 6-year-old boy with SLS to successfully obtain radiographic studies and extract a supernumerary tooth. No problems occurred other than difficulties with adhesives and soft tissue obstruction of the airway.
Dupilumab Reduces Pruritus in Twins With Sjögren-Larsson Syndrome.
Sjögren-Larsson Syndrome (SLS), now termed ALDH3A2-syndromic epidermal differentiation disorder (sEDD), is a rare genetic disorder marked by thickened skin, spasticity, and intellectual disability. Intractable pruritus is a nearly universal and debilitating feature of SLS that remains poorly managed by current therapies. We describe 4-year-old twin girls with genetically confirmed SLS who showed significant and lasting improvement in itch following treatment with dupilumab, a biologic targeting interleukin-4 receptor signaling. Within 6 months, pruritus severity scores declined markedly, improving sleep and quality of life without adverse effects, supporting further investigation of dupilumab for SLS-associated itch.
Ciliopathy: Sjögren-Larsson Syndrome.
Sjögren-Larsson syndrome is caused by a mutation in the ALDH3A2 gene, which produces fatty aldehyde dehydrogenase (FALDH). FALDH is a membrane-bound protein involved in fatty oxidation. Structural disturbance and poor metabolite clearance likely contribute to pathogenesis.
Biosynthesis of fatty aldehydes and alcohols in the eye and their role in meibogenesis.
Fatty alcohols (FAlc) and aldehydes (FAld) are essential intermediates/precursors in the biosynthesis of lipids. However, elevated FAld levels were shown to be geno- and cytotoxic, thus requiring conversion into less toxic FAlc and fatty acids (FA). An increase in FAlc and FAld in tissues of patients with Sjögren-Larsson syndrome was reported before and repeatedly linked to inactivation of ALDH3A2, which oxidizes FAld in FA. Recently, we hypothesized that another group of enzymes, namely SDR16C5/SDR16C6 (EC 1.1.1.105), could control the balance between FA, FAlc, and FAld via a separate mechanism. In this study, we assessed the in vivo biosynthesis of FAlc and FAld in mammals using Meibomian glands (MG) of wild-type (WT) and Sdr16c5/Sdr16c6-null (Hom) mice as models. Lipids were extracted from MG of experimental animals and analyzed using LC/MS. Because of high reactivity and instability of FAld, the compounds were initially converted to stable, sodium borohydride-reduced 3-aminopyridine conjugates, while FAlc were analyzed as N-alkyl pyridinium ions. A wide range of saturated and unsaturated FAld, FAlc, and FA ranging from C3 to C28 and longer were found in MG of mice of both genotypes. Our experiments revealed a multifold upregulation of almost all detected straight chain, but not branched, FAlc in MG lipidomes of Hom mice, which implied a previously unknown ability of SDR16C5/SDR16C6 to oxidize a wide range of FAlc in FAld in vivo. We have concluded that SDR16C5/SDR16C6 plays a central, and selective, role in FA/FAlc/FAld metabolism in vivo and proposed a generalized mechanism of these reactions.
Publicações recentes
Mutation mapping and functional characterization of a missense mutation p.Arg228Cys in ALDH3A2 gene causing Sjögran-Larson syndrome.
Multisystem manifestations of Sjögren-Larsson syndrome in early childhood and its dental implications.
General Anesthesia for a Child With Sjögren-Larsson Syndrome.
Dupilumab Reduces Pruritus in Twins With Sjögren-Larsson Syndrome.
Ciliopathy: Sjögren-Larsson Syndrome.
📚 EuropePMC326 artigos no totalmostrando 89
Multisystem manifestations of Sjögren-Larsson syndrome in early childhood and its dental implications.
BMJ case reportsGeneral Anesthesia for a Child With Sjögren-Larsson Syndrome.
Anesthesia progressDupilumab Reduces Pruritus in Twins With Sjögren-Larsson Syndrome.
Pediatric dermatologyCiliopathy: Sjögren-Larsson Syndrome.
Advances in experimental medicine and biologyBiosynthesis of fatty aldehydes and alcohols in the eye and their role in meibogenesis.
The Journal of biological chemistryAccumulation of ether phospholipids in induced pluripotent stem cells and oligodendrocyte-lineage cells established from patients with Sjögren-Larsson syndrome.
Congenital anomaliesDiscovery of novel diagnostic biomarkers for Sjögren-Larsson syndrome by untargeted lipidomics.
Biochimica et biophysica acta. Molecular and cell biology of lipidsCoexistence of Ichthyosis, Yellowish Keratoderma, and Neurologic Manifestations: Think About Sjogren-Larsson Syndrome.
SkinmedSjogren-Larsson syndrome brain volumetric reductions demonstrated with an automated software.
Arquivos de neuro-psiquiatriaUntargeted Metabolomic Analysis of Sjögren-Larsson Syndrome Reveals a Distinctive Pattern of Multiple Disrupted Biochemical Pathways.
MetabolitesEnd-stage crystalline maculopathy with retinal atrophy in Sjögren-Larsson syndrome: a case report and review of the literature.
Therapeutic advances in rare diseaseSjögren-Larsson Syndrome: A Rare Presentation With Developmental Delay.
CureusClinical and molecular characteristics of autosomal recessive congenital ichthyosis in Thailand.
Pediatric dermatologySjögren-Larsson syndrome caused by novel mutations in ALDH3A2 gene.
International journal of dermatologyCeramide profiling of stratum corneum in Sjögren-Larsson syndrome.
Journal of dermatological scienceSmall touches to big walks -the impact of rehabilitation on Sjögren-Larsson syndrome: A case report.
Journal of pediatric rehabilitation medicineBeyond retina in Sjogren-Larsson syndrome.
Indian journal of ophthalmologyRetinal Capillary Abnormalities in Sjögren-Larsson Syndrome Maculopathy.
Case reports in ophthalmologySjögren-Larsson syndrome: A biochemical rationale for using aldehyde-reactive therapeutic agents.
Molecular genetics and metabolism reportsImpaired Skin Barrier Function Due to Reduced ω-O-Acylceramide Levels in a Mouse Model of Sjögren-Larsson Syndrome.
Molecular and cellular biologySjögren-Larsson syndrome in Spain: Description of three new cases.
Anales de pediatriaA Neurodegenerative Phenotype Associated With Sjögren-Larsson Syndrome.
Journal of child neurologyNovel ALDH3A2 mutations in structural and functional domains of FALDH causing diverse clinical phenotypes in Sjögren-Larsson syndrome patients.
Human mutationMuscle Tone Assessment under General Anesthesia for Sjögren-Larsson Syndrome and Spasticity.
Pediatric neurosurgeryAldh1l2 knockout mouse metabolomics links the loss of the mitochondrial folate enzyme to deregulation of a lipid metabolism observed in rare human disorder.
Human genomicsSjögren-Larsson syndrome: Anesthetic considerations and practical recommendations.
Paediatric anaesthesiaSjogren-Larsson Syndrome: A case series of five members from an extended family with a novel mutation.
Molecular genetics & genomic medicine1-O-Alkylglycerol accumulation reveals abnormal ether glycerolipid metabolism in Sjögren-Larsson syndrome.
Molecular genetics and metabolismDisturbed brain ether lipid metabolism and histology in Sjögren-Larsson syndrome.
Journal of inherited metabolic diseaseMacular crystalline inclusions in Sjögren-Larsson syndrome are dynamic structures that undergo remodeling.
Ophthalmic geneticsSjögren-Larsson syndrome: The mild end of the phenotypic spectrum.
JIMD reportsComprehensive Molecular Profiles of Functionally Effective MSC-Derived Extracellular Vesicles in Immunomodulation.
Molecular therapy : the journal of the American Society of Gene TherapyComprehensive in silico screening and molecular dynamics studies of missense mutations in Sjogren-Larsson syndrome associated with the ALDH3A2 gene.
Advances in protein chemistry and structural biologySjogren-Larsson Syndrome: Mechanisms and Management.
The application of clinical geneticsNext-generation sequencing through multi-gene panel testing for diagnosis of hereditary ichthyosis in Chinese.
Clinical geneticsCompound heterozygous mutations in the ALDH3A2 gene cause Sjögren-Larsson syndrome: a case report.
The International journal of neuroscienceNovel imaging technologies for genetic diagnoses in the inborn errors of metabolism.
Journal of translational genetics and genomicsSjogren-Larsson syndrome associated hypermelanosis.
Journal of cosmetic dermatologySjögren-Larsson syndrome: a complex metabolic disease with a distinctive ocular phenotype.
Ophthalmic geneticsGenetic assessment of ten Egyptian patients with Sjögren-Larsson syndrome: expanding the clinical spectrum and reporting a novel ALDH3A2 mutation.
Archives of dermatological researchPhenotypic and mutational spectrum of thirty-five patients with Sjögren-Larsson syndrome: identification of eleven novel ALDH3A2 mutations and founder effects.
Journal of human geneticsRetinal Morphology in Sjögren-Larsson Syndrome on OCT: From Metabolic Crystalline Maculopathy to Early-Onset Macular Degeneration.
Ophthalmology. RetinaThe combination of whole-exome sequencing and clinical analysis allows better diagnosis of rare syndromic retinal dystrophies.
Acta ophthalmologicaDaily Functioning and Quality of Life in Patients with Sjögren-Larsson Syndrome.
Neuropediatrics[Sjögren-Larsson syndrome: Pediatric case report].
Archivos argentinos de pediatria[A new mutation in the ALDH3A2 gene in a boy with Sjogren-Larsson syndrome].
Revista de neurologiaGenotype and phenotype variability in Sjögren-Larsson syndrome.
Human mutationSjogren-Larsson Syndrome: A Rare Case Report.
Indian dermatology online journalLate-Stage Sjögren-Larsson Syndrome Maculopathy Imaged With OCT Angiography.
Ophthalmic surgery, lasers & imaging retinaNeurodegeneration in an adolescent with Sjogren-Larsson syndrome: a decade-long follow-up case report.
BMC medical geneticsNeural symptoms in a gene knockout mouse model of Sjögren-Larsson syndrome are associated with a decrease in 2-hydroxygalactosylceramide.
FASEB journal : official publication of the Federation of American Societies for Experimental BiologyNeuro-ichthyotic Syndromes: A Case Series.
Journal of pediatric neurosciencesTypical clinical and neuroimaging features in Sjögren-Larsson syndrome.
Arquivos de neuro-psiquiatriaClinical and molecular characterization and response to acitretin in three families with Sjögren-Larsson syndrome.
International journal of dermatologyIdentification of a novel deletion within ALDH3A2 gene in an Iranian Family with Sjögren-Larsson Syndrome.
Clinical case reportsSjögren-Larsson syndrome: definitive diagnosis on magnetic resonance spectroscopy.
CutisNovel mutations and a severe neurological phenotype in Sjögren-Larsson syndrome patients from Iran.
European journal of medical geneticsAtypical Presentation of Sjögren-Larsson Syndrome.
Case reports in pediatricsSequence variants in nine different genes underlying rare skin disorders in 10 consanguineous families.
International journal of dermatologyIdentification of Homozygous Likely Pathogenic Variant of ALDH3A2 in a Korean Boy with Sjögren-Larsson Syndrome.
Annals of laboratory medicineUnderstanding fetal factors that contribute to preterm birth: Sjögren-Larsson syndrome as a model.
Journal of perinatal medicineThe sphingosine 1-phosphate breakdown product, (2E)-hexadecenal, forms protein adducts and glutathione conjugates in vitro.
Journal of lipid researchClinical, biochemical, and genetic aspects of Sjögren-Larsson syndrome.
Clinical geneticsCoexistence of Two Rare Autosomal Recessive Disorders: Activation-Induced Cytidine Deaminase Deficiency and Sjogren-Larsson Syndrome.
The Israel Medical Association journal : IMAJExpanding the Genotype of Sjögren-Larsson Syndrome: A New Case Due to Two Novel Mutations.
Actas dermo-sifiliograficasLack of Long-Term Neurologic Efficacy of Zileuton in Sjögren-Larsson's Syndrome.
NeuropediatricsOPHTHALMIC FINDINGS IN LATE STAGE SJOGREN-LARSSON SYNDROME.
Retinal cases & brief reportsIntrathecal Baclofen Therapy for the Treatment of Spasticity in Sjögren-Larsson Syndrome.
Journal of child neurologyPNPLA1 is a transacylase essential for the generation of the skin barrier lipid ω-O-acylceramide.
Nature communications[Anesthesia for an Eleven Year Old Girl with Sjögren-Larsson Syndrome].
Masui. The Japanese journal of anesthesiologyChild Neurology: Sjögren-Larsson syndrome.
NeurologyUnusual presentation Of Sjögren-associated neuropathy with plasma cell-rich infiltrate.
Muscle & nerveMacular fibrosis complicating macular pigment deficient maculopathy in Sjögren-Larsson syndrome.
Acta ophthalmologicaGenetics and prospective therapeutic targets for Sjögren-Larsson Syndrome.
Expert opinion on orphan drugsA rare case of Sjogren-Larsson syndrome with recurrent pneumonia and asthma.
Korean journal of pediatricsSjögren-Larsson Syndrome: A Neuro-Ichthyotic Disorder With Unique Magnetic Resonance Features.
Pediatric neurologySjogren-Larsson syndrome: A rare neurocutaneous disorder.
Journal of pediatric neurosciencesSjögren-Larsson syndrome: a rare disease of the skin and central nervous system.
BMJ case reportsDisruption of the Sjögren-Larsson Syndrome Gene Aldh3a2 in Mice Increases Keratinocyte Growth and Retards Skin Barrier Recovery.
The Journal of biological chemistryInherited ichthyosis: Syndromic forms.
The Journal of dermatologyOcular manifestations of genetic skin disorders.
Clinics in dermatologySAXS fingerprints of aldehyde dehydrogenase oligomers.
Data in briefA rare cause of pruritic ichthyosis: Sjögren-Larsson syndrome in the first reported patients of Cypriot descent.
European journal of dermatology : EJDPhytol in a pharma-medico-stance.
Chemico-biological interactionsChanges in the Submandibular Salivary Gland Epithelial Cell Subpopulations During Progression of Sjögren's Syndrome-Like Disease in the NOD/ShiLtJ Mouse Model.
Anatomical record (Hoboken, N.J. : 2007)Zileuton for Pruritus in Sjögren-Larsson Syndrome: A Randomized Double-blind Placebo-controlled Crossover Trial.
Acta dermato-venereologicaCase of Sjögren-Larsson syndrome with a large deletion in the ALDH3A2 gene confirmed by single nucleotide polymorphism array analysis.
The Journal of dermatologySegmentation of Retinal Layers in Sjögren-Larsson Syndrome.
OphthalmologySingle amino acid polymorphism in aldehyde dehydrogenase gene superfamily.
Frontiers in bioscience (Landmark edition)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 Síndrome Sjögren-Larsson e no aconselhamento genético da família.
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
A condição é causada por alterações genéticas no gene ALDH3A2, responsável pela produção da enzima Aldehyde dehydrogenase family 3 member A2. Seu padrão de transmissão é autossômico recessivo, demandando uma cópia alterada herdada de cada genitor.
Referências
Fontes citadas no texto, publicações do grafo e bases de dados usadas neste verbete
6 publicações do grafo RarasNet (PubMed) · 8 bases de dados. Títulos, periódicos e PMIDs vêm direto da fonte, sem intermediação de IA.
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.). Síndrome Sjögren-Larsson. Em Raras — Enciclopédia de Doenças Raras do Brasil. https://raras.org/doenca/sindrome-sjogren-larsson
Formato APA. Conteúdo sob CC BY 4.0 — reuso livre com atribuição.
Conteúdo mantido por Agente Raras · Médicos e pesquisadores podem colaborar

Síndrome Sjögren-Larsson
📋 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
- Genética mendeliana
- fonte: OMIM
- Codificação WHO/SUS
- fonte: WHO ICD-10 / DATASUS
- CID-11 (futuro)
- fonte: WHO ICD-11
- NIH/GARD
- fonte: GARD (NIH)
- Indexação biomédica
- fonte: MeSH (NLM)
- Dado público estruturado
- fonte: Wikidata