Author name: Sarah McCall

Scholar Rock, a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for SRK-015 for the treatment of Spinal Muscular Atrophy (SMA), a progressive, rare genetic disease that […]

Dear SMA Patient Advocacy Community, As part of our ongoing partnership and following your request to receive updates about the risdiplam clinical development program, we are delighted to share with you a much-anticipated milestone. Today, the U.S. Food and Drug Administration (FDA) has approved risdiplam for the treatment of spinal muscular atrophy (SMA) in adults

Dear Members of the SMA Community, In light of the COVID-19 pandemic and the burden it has placed on our healthcare system, Biogen is working with health systems, institutions, physicians, patients and their families to navigate potential SPINRAZA® (nusinersen) dosing delays related to COVID-19. The health and safety of patients is our primary concern, and

This article is the last in a series of Cure SMA grant announcements that have been shared throughout the past months. Cure SMA has awarded a $190,000 research grant to Stephen Meriney, Ph.D., at the University of Pittsburgh for his project titled, “A novel treatment targeting persistent neuromuscular dysfunction in a mild mouse model of

Biogen today announced that the first patient has been treated in the global clinical study, DEVOTE. The study is designed to evaluate the safety, tolerability, and potential for even greater efficacy of Spinraza (nusinersen) when administered at a higher dose than currently approved for the treatment of spinal muscular atrophy (SMA). The Phase 2/3 randomized,

AveXis, a Novartis company, announced a one-time infusion of Zolgensma® (onasemnogene abeparvovec-xioi) showed rapid, significant, and clinically meaningful therapeutic benefit in patients with spinal muscular atrophy (SMA) across a range of studies, including in patients treated pre-symptomatically, and sustained durability in patients now up to 5 years post-dosing and some patients more than 5 years