Dear members of the SMA community, As we celebrate Rare Disease Day 2019, we are honored to continue supporting the spinal muscular atrophy (SMA) community. In recent weeks and months, we engaged the community on a number of different initiatives and are excited to share updates on our activities, support of new clinical research and

On Monday, March 11th, at 12pm CST (10am PST/11am MST/1pm EST) Genentech/Roche and Cure SMA will co-host a webinar for the SMA community. During the webinar, you’ll hear from Genentech/Roche on their history working with the SMA community, and their investigational medicine risdiplam. You’ll hear updates on their clinical trials in pre-symptomatic & Types 1,

Scholar Rock today announced positive interim results from the ongoing Phase 1 single- and multiple-ascending dose clinical trial of SRK-015 in healthy adult volunteers. SRK-015, a highly specific inhibitor of myostatin activation, was observed to be well tolerated with no apparent safety signals. By demonstrating robust and sustained target engagement in humans, pharmacologic effects on