Clinical trial highlights

SMA is a severe neuromuscular disease in which motor neuron degeneration may occur in the first months of life, often before symptoms appear. Risdiplam (EVRYSDI ™) is a centrally and peripherally distributed, oral survival of motor neuron 2 (SMN2) pre-mRNA splicing modifier that has been approved by the FDA for the treatment of patients with SMA, aged 2 months and older. RAINBOWFISH (NCT03779334) is an open-label, single-arm, multicenter study to investig ate efficacy, safety, and pharmacokinetics (PK)/pharmacodynamics of risdiplam in infants with genetically diagnosed presymptomatic SMA.
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research