Ana de Barros, PhD, managing science editor —

Cure SMA, an organization dedicated to the treatment and cure of spinal muscular atrophy (SMA), has launched a booklet addressing some of the broader questions about insurance coverage that are common to the SMA community. Decisions can seem overwhelming when faced with a new SMA diagnosis or when caring for…

In infants with spinal muscular atrophy (SMA), treatment with Spinraza (nusinersen), the first FDA-approved drug for SMA, reduces the risk of death or permanent ventilation, according to new results from the Phase 3 ENDEAR study (NCT02193074). Biogen presented the data at the British Paediatric Neurology Association (BPNA)…

Good Start Genetics and Roche Diagnostics have entered into a marketing agreement to sell Good Start’s GeneVu carrier screening service for inherited genetic disorders, such as cystic fibrosis (CF) and spinal muscular atrophy (SMA). Under the terms of the contract, Roche Diagnostics will provide obstetricians and general practitioners in the…

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…

AveXis reported that the planned pivotal clinical trial of its gene therapy candidate AVXS-101 for spinal muscular atrophy (SMA) Type 1 will enroll about 20 patients and will have a single-arm design, with the comparator being the natural disease history. This update follows the Type B meeting Sept. 30 with the…

Biogen has completed its rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of nusinersen for the treatment for spinal muscular atrophy (SMA). Biogen has also applied for a Priority Review designation which, if granted, means the FDA should…

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, 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…

The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to AVXS-101, AveXis’ lead development candidate to treat spinal muscular atrophy (SMA) Type 1 in pediatric patients. AVXS-101 is a proprietary gene therapy candidate of a one-time, intravenous treatment for SMA Type 1, and, according to AveXis, is the…

Cure SMA recently granted a $304,000 drug discovery award to Livio Pellizzoni, assistant professor of Pathology & Cell Biology at Columbia University for a new research project with potential to treat spinal muscular atrophy (SMA). Pellizzoni and collaborators at Northwestern University have identified an altered cellular pathway in SMA patients, which may…