Author Archives: Ana de Barros PhD

Spinraza Meets Primary Endpoint for Later-onset SMA in Phase 3 Trial

Spinraza (nusinersen) for spinal muscular atrophy (SMA) met the primary endpoint in an interim analysis from a Phase 3 clinical trial evaluating the treatment in children with later-onset (consistent with Type 2) SMA. Biogen and Ionis Pharmaceuticals reported that children treated with Spinraza had a highly statistically significant improvement in motor…

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…

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…

AveXis Releases Favorable Data in Ongoing Phase 1 Test of SMA Gene Therapy, AVXS-101

AveXis recently reported favorable results from an ongoing Phase 1 clinical trial examining its candidate, AVXS-101, for the systematic delivery of gene therapy to young patients with spinal muscular atrophy (SMA). These preliminary results will help inform upcoming trials on gene therapy approaches. The open-label, dose-escalation study evaluating the safety and efficacy of…