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3. Spinal muscular atrophy Disease details / Clinical trials / Drug dev / DR info

Clinical trials : 297 / Drugs : 143 - (DrugBank : 32) / Drug target genes : 54 - Drug target pathways : 82

Other names

SMA;Myelopathic muscular atrophy;Werdnig-Hoffman disease;Dubowitz disease;Kugelberg-Welander disease;

Disease group

Neuromuscular diseases

Domestic patients
Med expenses recipients (FY2023)

955 patients

Age distribution

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Related info (in Japanese)

Clinical research form [April 1, 2025 ~] (in Japanese)

Overview, diagnostic criteria, etc. (pdf), Clinical research form (pdf)

Specific pediatric chronic diseases, Japan (in Japanese)

11-19-44. spinal muscular atrophy; SMA

Subtypes

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