SMA Press
Roche to begin Part 2 of the SUNFISH Trial
To read the full press release, please click here: SUNFISH
Read MoreScholar Rock to Develop SRK-015 to Improve Muscle Function in Patients with Spinal Muscular Atrophy
First data on inhibition of myostatin activation in a preclinical model of SMA to be highlighted at the Cure SMA Annual Conference Treatment with SRK-015 resulted in significantly increased muscle strength in a genetic model of SMA CAMBRIDGE, Mass., June 28, 2017 – Scholar Rock, a biotechnology company focused on discovering and developing drugs that…
Read MoreRG7916 Granted Orphan Drug Designation in the U.S. for the Treatment of Spinal Muscular Atrophy
SOUTH PLAINFIELD, N.J., January 6, 2017 – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (OOD) to RG7916 for the treatment of patients with Spinal Muscular Atrophy (SMA). RG7916 is part of PTC’s joint development program in SMA with Roche and the SMA…
Read MorePTC Therapeutics Announces Initiation of FIREFISH Study in Infant (Type I) SMA Patients
SOUTH PLAINFIELD, N.J., January 5, 2017 – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that its joint development program in Spinal Muscular Atrophy (SMA) with Roche and the SMA Foundation (SMAF) initiated a clinical study in infants with Type I SMA. The study, named FIREFISH, will investigate the safety, tolerability and efficacy of RG7916 in…
Read MoreFDA Approves Biogen’s SPINRAZA™ (nusinersen) for Spinal Muscular Atrophy
The SMA Foundation is excited to share the news that the U.S. Food and Drug Administration (FDA) approved Biogen’s SPINRAZA™ (nusinersen) under Priority Review for the treatment of Spinal Muscular Atrophy (SMA) in pediatric and adult patients. SPINRAZA is the first and only treatment approved for SMA. (Please see http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm534611.htm for additional information.) SPINRAZA offers…
Read MoreComprehensive Book on Spinal Muscular Atrophy Published
We are pleased to announce that the book “Spinal Muscular Atrophy: Disease Mechanisms and Therapy,” has been published by Elsevier and is now available in retail outlets, as well as at the Elsevier Store in print and electronic formats here. This book is a comprehensive reference of recent advances in basic and clinical SMA research…
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