SMA treatment Spinraza followed by Zolgensma improved motor function and prevented worsening of breathing difficulties in a ...

Based on a small trial with positive muscle performance data, Regenxbio is expected to pursue approval of its gene therapy ...

Roche’s Evrysdi (risdiplam) is the market leader among the drugs for spinal muscular atrophy (SMA). Biogen’s Spinraza ...

Karnataka launched free treatment for Spinal Muscular Atrophy (SMA) at the Indira Gandhi Institute of Child Health in ...

Alpana Sharma, founder-director of CureSMA, a parent-led advocacy group, urges the government to include medicines for SMA in ...

These updates will also be presented at the American Society of Gene and Cell Therapy and Muscular Dystrophy ... including Novartis' Zolgensma ® for children with spinal muscular atrophy. Designed to ...

Muscle fibers in children with SMA type 1 show substantial differences in components of the myosin protein called heavy ...