February 14, 2018
Biogen has provided the following community statement on the final results from CHERISH, a Phase 3 study of SPINRAZA.
Dear Members of the SMA community,
This past December marked one year since the FDA approval of SPINRAZA® (nusinersen), the first and only treatment...
February 1, 2018
AveXis has provided the following community statement on AVXS-101.
Dear SMA Community,
AveXis, the gene therapy company developing a new approach to treat SMA known as AVXS‐101, is pleased to initiate screening for the remaining patients to be enrolled in the pivotal trial of...
January 30, 2018
Preliminary Data from FIREFISH Trial in Type 1 SMA Infants Presented at the International Scientific Congress on Spinal Muscular Atrophy
PTC Therapeutics, Inc. recently announced the presentation of early interim data from Part 1, the dose-finding portion of the FIREFISH study. FIREFISH is a two-part seamless, open-label, multi-center study to investigate the safety and efficacy of RG7916 in infants and babies with Type 1 SMA. RG7916 has been safe and well tolerated at all...