SMA is usually categorized by a pattern of weakness whether predominantly proximal or distal and mode of inheritance. Spinal muscular atrophy type 0 is evident before birth and is the rarest and ...

San Diego: Academic Press; 2015:117-145. Lefebvre S, Burglen L, Reboullet S, et al. Identification and characterization of a spinal muscular atrophy-determining gene. Cell.1995;80 (1):155-165.

Spinal muscular atrophy varies in severity. The worst and most common form, called type 1, usually leads to death within the first two to four years of life. Less severe types can cause lifelong ...

Manon Nadeau, whose 24-year-old son has Type 3 spinal muscular atrophy, said she cried when she learned about the funding. The drug, Spinraza, costs $118,000 per dose and a minimum of three doses ...

Bray has spinal muscular atrophy, a rare degenerative disease that's gradually robbed him of the ability to move his muscles. At age 29, he can no longer move his head, arms, hands or legs.

Doctors at St. Jude’s Research Hospital announced that a patient with spinal muscular atrophy shows no signs of the rare genetic disorder after receiving the first-ever treatment in the womb.

Novartis has reported positive topline outcomes from the Phase III STEER trial of an investigational gene therapy, intrathecal onasemnogene abeparvovec (OAV101 IT) in treating individuals with ...

NIDO-361, our clinical-stage candidate, is a treatment for Spinal and Bulbar Muscular Atrophy which is a rare and debilitating neuromuscular disease.