A doença de Kennedy, também conhecida como atrofia muscular bulboespinhal (BSMA), é uma doença rara do neurônio motor recessivo ligada ao X, caracterizada por perda de músculo proximal e bulbar.
Introdução
O que você precisa saber de cara
A doença de Kennedy, também conhecida como atrofia muscular bulboespinhal (BSMA), é uma doença rara do neurônio motor recessivo ligada ao X, caracterizada por perda de músculo proximal e bulbar.
Tem tratamento?
Escala de raridade
<1/50kMuito rara
1/20kRara
1/10kPouco freq.
1/5kIncomum
1/2k
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Entender a doença
Do básico ao detalhe, leia no seu ritmo
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Sinais e sintomas
O que aparece no corpo e com que frequência cada sintoma acontece
Partes do corpo afetadas
+ 13 sintomas em outras categorias
Características mais comuns
Os sintomas variam de pessoa para pessoa. Abaixo estão as 28 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: X-linked recessive.
Steroid hormone receptors are ligand-activated transcription factors that regulate eukaryotic gene expression and affect cellular proliferation and differentiation in target tissues (PubMed:19022849). Transcription factor activity is modulated by bound coactivator and corepressor proteins like ZBTB7A that recruits NCOR1 and NCOR2 to the androgen response elements/ARE on target genes, negatively regulating androgen receptor signaling and androgen-induced cell proliferation (PubMed:20812024). Tran
NucleusCytoplasm
Androgen insensitivity syndrome
An X-linked recessive form of pseudohermaphroditism due end-organ resistance to androgen. Affected males have female external genitalia, female breast development, blind vagina, absent uterus and female adnexa, and abdominal or inguinal testes, despite a normal 46,XY karyotype.
Medicamentos e terapias
Mecanismo: Steroid 5-alpha-reductase inhibitor
Mecanismo: Beta-2 adrenergic receptor agonist
Variantes genéticas (ClinVar)
569 variantes patogênicas registradas no ClinVar.
Classificação de variantes (ClinVar)
Distribuição de 667 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 — Doença de Kennedy
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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
Ensaios em destaque
🟢 Recrutando agora
1 pesquisa recrutando participantes. Converse com seu médico sobre a possibilidade de participar.
Outros ensaios clínicos
211 ensaios clínicos encontrados, 1 ativos.
Publicações mais relevantes
[Perioperative management of a patient with Kennedy disease undergoing knee replacement: A case report].
Kennedy disease is a rare X-linked recessive genetic disease with a low incidence rate. The main manifestations of motor neuron involvement include limb weakness, muscle atrophy, dysarthria, difficulty swallowing, and coughing after drinking water. The patient may die from pulmonary infection and respiratory failure, and there is currently no effective treatment available. There are few reports on anesthesia for such patients and no guidelines or expert consensus. This article reports on perioperative anesthesia management for a 69 years old patient who underwent lumbar spine surgery before with coronary heart disease. The patient was diagnosed with Kennedy disease through electromyography and genetic testing before surgery and underwent knee replacement surgery. After sufficient preoperative consultation and evaluation, femoral nerve block was performed with 0.25% ropivacaine under guidance with ultrasound and nerve stimulator, followed by induction of general anesthesia with sufentanil, propofol, and etomidate. A laryngeal mask was inserted without the use of muscle relaxants and breathing was controlled by machine. During the operation, propofol and remifentanil were used for total intravenous anesthesia. The patient had stable vital signs, well tolerated, and the surgical process was smooth. The time of recovery from anesthesia was short, and no anesthesia related complications, such as nausea, vomiting, aspiration, or suffocation was observed after the operation. Postoperative muscle strength recovery was good. After closely monitoring in the ICU for a day, the patient returned to the regular ward. A postoperative analgesia combination of nerve block and oral nonsteroidal analgesics was performed, and emergency pain rescue with pethidine was administered if necessary. The analgesic effect was satisfactory. The patient was safely discharged in the end and recovered well. 肯尼迪病是一种罕见的X染色体连锁隐性遗传疾病, 发病率低, 以下运动神经元受累为主要表现, 具体可表现为四肢无力、肌肉萎缩、构音障碍、吞咽困难、饮水呛咳等。患者可能死于肺部感染和呼吸衰竭, 目前尚无有效治疗手段。此病患者临床麻醉学报道较少见, 并无明确的指南或专家共识。本文报道1例69岁合并冠心病的腰椎术后患者, 术前经肌电图检查和基因检测确诊为肯尼迪病, 行膝关节置换术的围术期麻醉处理过程。经过充分的术前会诊和评估, 在超声和神经刺激器引导下以0.25%(质量分数)罗哌卡因行股神经阻滞后, 以舒芬太尼、丙泊酚和依托咪酯进行全身诱导, 在不使用肌松剂的情况下置入喉罩, 控制呼吸。术中采用丙泊酚和瑞芬太尼全凭静脉麻醉, 患者生命体征平稳, 耐受良好, 手术过程顺利。术后患者苏醒迅速, 未出现恶心、呕吐、误吸、窒息等麻醉相关并发症, 术后肌力恢复良好, 于重症监护病房密切监测1 d后, 返回普通病房。术后采用神经阻滞联合口服非甾体类镇痛药, 必要时予哌替丁紧急补救的镇痛方案, 镇痛效果满意, 最终患者安全出院, 愈后良好。
A Dual Setback: Staphylococcus aureus Biofilms From Severe Chronic Rhinosinusitis Show Enhanced Virulence Capacity and Tolerance to Antibiotics.
Staphylococcus aureus biofilms play a crucial role in chronic rhinosinusitis (CRS), leading to the persistence of symptoms. Severe CRS patients are frequently infected with S. aureus strains that exhibit higher biofilm properties (e.g., biomass, exoprotein production) compared to S. aureus from controls. S. aureus biofilms resist antibiotic treatment; however, the relationship between bacterial biofilm properties, antibiotic susceptibility, and CRS severity has not yet been defined and is the subject of this study. S. aureus clinical isolates and reference strains, and matched clinical datasets were collected from CRS patients and controls (n = 35). Antimicrobial susceptibility of the isolates to clindamycin, mupirocin, clarithromycin, doxycycline, and amoxicillin-clavulanic acid was determined by minimum inhibitory concentration (MIC) and minimum biofilm eradication concentration (MBEC). S. aureus MBEC values (n = 35) were significantly higher (up to 11 times) than the MIC for all five antibiotics (p < 0.001). Among the various antibiotics tested, mupirocin had the strongest antibiofilm activity and amoxicillin-clavulanic acid the weakest: at antibiotic concentrations that are deemed to indicate susceptibility or intermediate resistance when testing in planktonic form, 80% biofilm eradication was reached for all isolates using mupirocin and only 5/35 isolates using amoxicillin-clavulanic acid. The biofilm metabolic activity, biomass, colony-forming units, and exoprotein production were positively correlated with the MBEC values for amoxicillin-clavulanic acid and clarithromycin, but not for the other antibiotics. Lund-Mackay and Lund-Kennedy disease severity scores showed positive correlations with the MBEC values of clarithromycin. These findings show that whilst severe CRS patients are frequently infected with S. aureus strains that exhibit higher biofilm-mediated virulence, these biofilms are also more difficult to control with standard of care antibiotics. Better personalized therapies are required to manage biofilm-mediated infections in severe CRS patients.
Exosome-rich mesenchymal stem cell secretome improves strength in patients with amyotrophic lateral sclerosis, Kennedy disease, congenital myasthenic syndrome and Lewy body dementia.
Amyotrophic lateral sclerosis (ALS), Lewy Body dementia (LBD), Kennedy disease (KD), and Congenital Myasthenic Syndrome (CMS) are progressive motor disorders for which no disease modifying treatment exists. ALS and LBD are uniformly, and often rapidly, fatal. No treatment of any kind has ever resulted in actual improvement for ALS patients; the best that has been achieved is minor slowing of their progression. Forty-one preclinical studies of intra-nasal instillation of mesenchymal stem cell exosomes have, however, demonstrated complete safety and efficacy for models of a variety of neurocognitive and motor disorders. We hypothesized that intranasal exosomes treatment in humans would be completely safe and also effective for the treatment of motor disorders such as ALS, LBD, KD and CMS. 18 patients with ALS, Kennedy Disease, Congenital Myasthenic Syndrome, or Lewy Body Dementia had 32 AlloEx Exosome® treatments to assess safety, attenuation of disease, and increase in strength and motor function. The study was conducted under the clinical trial NCT07105371 found at clinicaltrials.gov/study/NCT07105371. There were no adverse events of any kind reported among these treatments. All patients, except for one, achieved some degree of clinical and strength improvement; the longest improvement was recorded at the 6-month follow-up. Intranasally-instilled AlloEx Exosomes® are completely safe, attenuate progression, and improve strength in ALS, Kennedy Disease, CMS, and LBD.
Spinal and bulbar muscular atrophy with hand tremors and chronic limb weakness, Kennedy disease.
Spinal and bulbar muscular atrophy (SBMA) is an X-linked neuromuscular disorder primarily affecting adult males due to the expansion of CAG repeats in the androgen receptor gene. It manifests as progressive lower motor neuropathy and androgen deficiency. A Japanese man in his late 50s presented with gradually progressive muscle weakness over 6 years. Examination revealed muscle weakness and atrophy in upper and lower limbs, decreased deep tendon reflexes, involuntary facial movements, bilateral finger tremors, tongue atrophy, fasciculations and bilateral gynaecomastia. Blood tests indicated elevated creatine kinase and mild hepatic dysfunction. Nerve conduction studies showed decreased sensory nerve action potentials, and electromyography demonstrated neurogenic changes. Genetic testing confirmed SBMA with 47 CAG repeats despite no family history. Treatment included leuprorelin acetate and rehabilitation using a wearable cyborg hybrid-assistive limb. As SBMA progresses slowly and symptoms like hand tremors and decreased serum creatinine precede significant weakness, early recognition is critical for diagnosis.
Hearing Function in Spinal and Bulbar Muscular Atrophy (SBMA): A Case Control Study From a Tertiary Referral Center.
Expanding on earlier findings of auditory involvement from two small-scale studies, we conducted a comprehensive evaluation of hearing levels in a larger cohort of SBMA patients. Thirty-six SBMA patients and 36 age-matched male controls without risk factors for hearing loss underwent a comprehensive audiological assessment, including pure-tone audiometry at 250, 500, 1000, 2000, 4000, and 8000 Hz frequencies, using both air and bone conduction. The pure-tone average (PTA) was calculated as the mean threshold at 500, 1000, 2000, and 4000 Hz. A correlation analysis was performed to evaluate the relationship between patients' audiological features and clinical characteristics, including motor disability, as measured by the SBMA functional rating scale (SBMAFRS) and the 6-min walk test (6MWT). PTA values were significantly higher in SBMA patients compared to healthy controls (Mann-Whitney U test, p = 0.0005, and p = 0.0001 for the right and left side, respectively), even when analysis was restricted to the 19 SBMA patients without risk factors for hearing loss (Mann-Whitney U test, p = 0.0148 and p = 0.0243 for the right and left ear, respectively). In the latter group, the hearing thresholds of each individual frequency were significantly higher than in controls, except for the intermediate frequencies (2000 Hz on both sides and 1000 on the left one). Negative significant correlations were found between PTA values and both the CAG repeat number and 6MWT distances. Conversely, SBMAFRS scores were overall unrelated to PTA values. Our findings suggest a disease-specific hearing impairment in SBMA patients.
Publicações recentes
[Clinical characteristics and genetic analysis of a patient with Kennedy disease with secondary infertility as the initial symptom].
[Perioperative management of a patient with Kennedy disease undergoing knee replacement: A case report].
A Dual Setback: Staphylococcus aureus Biofilms From Severe Chronic Rhinosinusitis Show Enhanced Virulence Capacity and Tolerance to Antibiotics.
Exosome-rich mesenchymal stem cell secretome improves strength in patients with amyotrophic lateral sclerosis, Kennedy disease, congenital myasthenic syndrome and Lewy body dementia.
Spinal and bulbar muscular atrophy with hand tremors and chronic limb weakness, Kennedy disease.
📚 EuropePMC75 artigos no totalmostrando 51
[Clinical characteristics and genetic analysis of a patient with Kennedy disease with secondary infertility as the initial symptom].
Zhonghua yi xue yi chuan xue za zhi = Zhonghua yixue yichuanxue zazhi = Chinese journal of medical genetics[Perioperative management of a patient with Kennedy disease undergoing knee replacement: A case report].
Beijing da xue xue bao. Yi xue ban = Journal of Peking University. Health sciencesA Dual Setback: Staphylococcus aureus Biofilms From Severe Chronic Rhinosinusitis Show Enhanced Virulence Capacity and Tolerance to Antibiotics.
International forum of allergy & rhinologyExosome-rich mesenchymal stem cell secretome improves strength in patients with amyotrophic lateral sclerosis, Kennedy disease, congenital myasthenic syndrome and Lewy body dementia.
American journal of stem cellsSpinal and bulbar muscular atrophy with hand tremors and chronic limb weakness, Kennedy disease.
BMJ case reportsThe Insufficient Number of Informative SNPs in a Preclinical Karyomapping Test for PGT-M Depends on the Reference Selected.
Journal of personalized medicineHearing Function in Spinal and Bulbar Muscular Atrophy (SBMA): A Case Control Study From a Tertiary Referral Center.
European journal of neurologyKennedy Disease in Latin America: Two New Mexican Cases and a Systematic Review of Regional Reports.
CureusGeneration of an induced pluripotent stem cell line from a Kennedy Disease patient with AR mutation.
Stem cell researchGeneration of an induced pluripotent stem cell (iPSC) line (INNDSUi007-A) from a patient with Kennedy disease.
Stem cell researchCAGn Polymorphic Locus of Androgen Receptor (AR) Gene in Russian Infertile and Fertile Men.
International journal of molecular sciencesOne case of spinal bulbar muscular atrophy misdiagnosed as polymyositis: Case report.
Medicine271st ENMC international workshop: Towards a unifying effort to fight Kennedy's disease. 20-22 October 2023, Hoofddorp, Netherlands.
Neuromuscular disorders : NMDDouble trouble: Kennedy Disease and Immune-Mediated Necrotizing Myopathy in a Cree Male.
The Canadian journal of neurological sciences. Le journal canadien des sciences neurologiquesClinical spectrum, biochemical profile and disease progression of Kennedy disease in an Indian cohort.
Internal medicine journalGenotype and clinical phenotype analysis of a Family with Kennedy disease.
MedicineDifferentiation of Motor Speech Disorders through the Seven Deviance Scores from MonPaGe-2.0.s.
Brain sciencesProximal Dominant Hereditary Motor and Sensory Neuropathy with TFG Mutation: First Case Report from India.
Neurology IndiaMild Androgen Insensitivity Syndrome: The Current Landscape.
Endocrine practice : official journal of the American College of Endocrinology and the American Association of Clinical EndocrinologistsCharacteristics of spinal and bulbar muscular atrophy in South Korea: a cross-sectional study of 157 patients.
Brain : a journal of neurologyApplication Value of the Motor Unit Number Index in Patients With Kennedy Disease.
Frontiers in neurologyKennedy disease in two sisters with biallelic CAG expansions of the androgen receptor gene.
Neuromuscular disorders : NMDClinical Phenotyping and Biomarkers in Spinal and Bulbar Muscular Atrophy.
Frontiers in neurologyThe prevalence of hereditary neuromuscular disorders in Northern Norway.
Brain and behaviorBulbospinal muscular atrophy (Kennedy disease) responsive to immunoglobulins?
Clinical case reportsThe French national protocol for Kennedy's disease (SBMA): consensus diagnostic and management recommendations.
Orphanet journal of rare diseasesMonozygotic Twins Discordant for Kennedy Disease: A Case Report.
Journal of clinical neuromuscular diseaseBright tongue sign in Kennedy disease.
QJM : monthly journal of the Association of PhysiciansAmyotrophic lateral sclerosis due to a SOD1 mutation and X-linked recessive bulbospinal neuronopathy in a single family.
Acta neurologica BelgicaThe black pearl of the ocean: Kennedy disease.
Neurology IndiaMotor Neuron Disease: Pathophysiology, Diagnosis, and Management.
The American journal of medicineOnly some patients with bulbar and spinal muscular atrophy may develop cardiac disease.
Molecular genetics and metabolism reportsPerioral and tongue fasciculations in Kennedy's disease.
Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical NeurophysiologyA severe phenotype of Kennedy disease associated with a very large CAG repeat expansion.
Muscle & nervePatient-identified impact of symptoms in spinal and bulbar muscular atrophy.
Muscle & nerveKennedy disease with difficulty in differential diagnosis: A case report.
MedicineKennedy disease (X-linked recessive bulbospinal neuronopathy): A comprehensive review from pathophysiology to therapy.
Revue neurologiqueAdult-onset spinal muscular atrophy: An update.
Revue neurologiqueA population-based epidemiologic study of adult neuromuscular disease in the Republic of Ireland.
NeurologySequence Context Influences the Structure and Aggregation Behavior of a PolyQ Tract.
Biophysical journalVery late-onset Sandhoff disease presenting as Kennedy disease.
Muscle & nerveTowards a European Registry and Biorepository for Patients with Spinal and Bulbar Muscular Atrophy.
Journal of molecular neuroscience : MNKennedy Disease Misdiagnosed as Polyneuropathy, Organomegaly, Endocrinopathy, Monoclonal Gammopathy, and Skin Changes (POEMS) Syndrome: A Case Report.
Medical principles and practice : international journal of the Kuwait University, Health Science CentreExercise Therapy in Spinobulbar Muscular Atrophy and Other Neuromuscular Disorders.
Journal of molecular neuroscience : MNClinical Trials in Spinal and Bulbar Muscular Atrophy-Past, Present, and Future.
Journal of molecular neuroscience : MNSpinal and Bulbar Muscular Atrophy.
Neurologic clinicsMitochondrial implications in bulbospinal muscular atrophy (Kennedy disease).
Amyotrophic lateral sclerosis & frontotemporal degeneration[Upper motor neuron involvement in Kennedy disease evaluated by triple stimulation technique].
Zhonghua yi xue za zhiTeaching video NeuroImages: hand tremor, tongue and perioral fasciculation in a patient with Kennedy disease.
NeurologyLaboratory evaluation of suspected motor neuron disease: A survey of physicians.
Muscle & nerveContractile dysfunction in muscle may underlie androgen-dependent motor dysfunction in spinal bulbar muscular atrophy.
Journal of applied physiology (Bethesda, Md. : 1985)Associações
Organizações que acompanham esta doença — pra ter apoio e orientação
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Comunidades
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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.
- [Perioperative management of a patient with Kennedy disease undergoing knee replacement: A case report].Beijing da xue xue bao. Yi xue ban = Journal of Peking University. Health sciences· 2026· PMID 41633609mais citado
- A Dual Setback: Staphylococcus aureus Biofilms From Severe Chronic Rhinosinusitis Show Enhanced Virulence Capacity and Tolerance to Antibiotics.
- Exosome-rich mesenchymal stem cell secretome improves strength in patients with amyotrophic lateral sclerosis, Kennedy disease, congenital myasthenic syndrome and Lewy body dementia.
- Spinal and bulbar muscular atrophy with hand tremors and chronic limb weakness, Kennedy disease.
- Hearing Function in Spinal and Bulbar Muscular Atrophy (SBMA): A Case Control Study From a Tertiary Referral Center.
- [Clinical characteristics and genetic analysis of a patient with Kennedy disease with secondary infertility as the initial symptom].
Bases de dados e fontes oficiais
Identificadores e referências canônicas usadas para montar este verbete.
- ORPHA:481(Orphanet)
- OMIM OMIM:313200(OMIM)
- MONDO:0010735(MONDO)
- GARD:6818(GARD (NIH))
- Variantes catalogadas(ClinVar)
- Busca completa no PubMed(PubMed)
- Q1995327(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
