The below statement is a community update from Scholar Rock on SRK-015.  Dear SMA Community, Scholar Rock is dedicated to developing novel medicines to improve the lives of individuals who have Spinal Muscular Atrophy (SMA). We want to thank the SMA community for your continued support of our mission. We are excited to share that

Genentech, a member of the Roche Group, recently announced new data at the 71st American Academy of Neurology (AAN) Annual Meeting from the dose-finding Part 1 of the pivotal FIREFISH trial showing infants with Type 1 spinal muscular atrophy (SMA) achieved key motor milestones after one year of treatment with investigational risdiplam. Among the infants

AveXis, a Novartis company, recently announced interim data from ongoing trials of the investigational product Zolgensma® (onasemnogene abeparvovec-xioi; AVXS-101) that showed positive results across a broad spectrum of patients with spinal muscular atrophy (SMA). These included the first presentation of data from the Phase 1 STRONG trial, which showed motor function gains and milestone achievements

Biogen today announced it will present new data focusing on SMA at the 71st annual meeting of the American Academy of Neurology (AAN) in Philadelphia, PA., May 4–11. Results from the NURTURE study demonstrate that pre-symptomatic infants with SMA treated with SPINRAZA over three years achieved motor milestones more consistent with normal childhood development. After

Genentech, a member of the Roche Group, announced today that new data will be presented at the 71st American Academy of Neurology (AAN) Annual Meeting from May 4-10 in Philadelphia, PA. Presentations include data from a pivotal study for risdiplam in spinal muscular atrophy (SMA), which has the potential to become the first oral treatment