Global Risdiplam Compassionate Use Program for Patients With Type 1 or 2 Spinal Muscular Atrophy
Description
PURPOSE: Spinal muscular atrophy (SMA) is a genetic neuromuscular disease causing progressive muscle weakness and reducing life expectancy. Risdiplam (Evrysdi; Genentech/F. Hoffmann-La Roche Ltd, Basel, Switzerland) is a drug approved for use in the
