Distal Spinal Muscular Atrophy Type 3

Watchlist
Retrieved
2021-01-23
Source
Trials
Genes
Drugs

Distal spinal muscular atrophy type 3 is a rare neuromuscular disease characterized by progressive muscular weakness and atrophy predominantly affecting distal parts of limbs, later involvement of proximal and trunk muscles with marked hyperlordosis and late diaphragmatic dysfunction.