Spinal muscular atrophy, or SMA, is a rare, genetic neuromuscular disease characterized by the loss of motor neurons, atrophy of the voluntary muscles of the limbs and trunk, and progressive muscle weakness. An estimated 30,000 to 35,000 people in the United States and Europe are afflicted by SMA. Based on the age and severity of symptoms, SMA is classified into 4 types namely, types 1 through 4.
The company we are profiling today is developing a drug for nonambulatory patients with Types 2 and 3 spinal muscular atrophy - Scholar Rock Holding Corp. SRRK).
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