The Canadian Institutes for Health Research, a federal government agency based in Ottawa, has awarded genetics professor Kessen Patten C$627,300 to study the mechanisms involved in spinal muscular atrophy (SMA). The leading genetic cause of infant mortality, SMA is a neuromuscular disease that involves the death of lower motor neurons — the…
AveXis is working with the U.S. Food and Drug Administration (FDA) to overcome final regulatory hurdles before starting a pivotal trial of AVXS-101 for the treatment of spinal muscular atrophy type 1 (SMA type 1) later in 2017. AVXS-101 is a gene therapy for SMA that has shown…
A crucial two-hour meeting between AveXis executives and officials of the U.S. Food and Drug Administration (FDA) will determine the next step for AVXS-101, a spinal muscular atrophy (SMA) treatment that has shown remarkable promise in a Phase 1 trial of babies with SMA type 1 — the most severe form of this muscle-damaging disease.
Spinraza (nusinersen), a treatment for most forms of spinal muscular atrophy (SMA), took a major step forward to becoming available across the European Union, with the Committee for Medicinal Products for Human Use (CHMP) recommending its approval. CHMP is a branch of the European Medicines Agency (EMA), the equivalent of…
A meeting that the spinal muscular atrophy (SMA) community is having with Food and Drug Administration officials in the Washington area next week is full, but will be live-streamed. The gathering on April 18 in Hyattsville, Maryland, will focus on the development of therapies for SMA. Patients, their families and caregivers…
In a recent webinar on Spinraza, the first approved treatment for spinal muscular atrophy (SMA), the patient advocacy group Cure SMA addressed issues such as access and insurance coverage, and answered questions from people affected by the disease. Cure SMA, a foundation that promotes SMA research and provides support for patients and their…
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