News

Therapy for Infantile-Onset SMA, Nusinersen, Shows Promise in Phase 3 Study

Biogen and Ionis Pharmaceuticals recently reported that nusinersen, an antisense drug designed to treat spinal muscular atrophy (SMA), ably met the primary endpoint set for an interim analysis of its Phase 3 ENDEAR clinical trial. That study, evaluating nusinersen as a treatment for infantile-onset (consistent with Type 1) SMA, is now closing and the program expanding…

Cure SMA to Put $2.5 Million Toward Research Efforts

Cure SMA, an organization dedicated to the treatment and cure of Spinal Muscular Atrophy (SMA), will provide $2.5 million over the next 12 months for research and to ensure the development of therapies for all ages, types, and stages of SMA. The announcement was made during the organization’s 2016 Annual SMA…

Clinical Development for SMA Treatments Reviewed at Annual Conference

Progress reports on six treatment options for spinal muscular atrophy (SMA), all currently in clinical trials, were spotlighted at the recent 2016 Annual SMA Conference in Anaheim, CA. The updates, provided by study representatives, shows that efforts are advancing toward new therapies for SMA. Four of the six treatment programs — AVXS-101, nusinersen, RG7800/RG7916, and…