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A new neonatal screening test recently saved the life of a little girl with spinal muscular atrophy, a rare neuromuscular ...
Learn what you need to do to have a fun, safe, and stress-free day at an amusement park when you have spinal muscular atrophy.
Objective The aim of this study was to assess early language acquisitions in treated individuals with spinal muscular atrophy (SMA) type 1 and in infants identified by newborn screening (NBS). Methods ...
Treatment with salanersen slowed neurodegeneration and improved motor function in children with SMA in a Phase 1 trial, ...