— First Reported Fast Track Designation for a FSHD Treatment — SAN DIEGO, Oct. 24, 2016 /PRNewswire/ — aTyr Pharma, Inc. (Nasdaq: LIFE), a biotherapeutics company engaged in the discovery and… Read More »
aTyr Pharma Receives FDA Fast Track Designation for Resolaris™
Acceleron to host webcast on FSHD clinical trial
(Via BusinessWire.) Acceleron Pharma Inc., a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of innovative therapeutics to treat serious and rare diseases, today announced that it… Read More »
Fruit fly model of FSHD is now available
The laboratory of Peter Jones at the University of Massachusetts Medical School reports that Drosophila (fruit fly) lines for studying DUX4 and FRG1 overexpression are freely available worldwide from the… Read More »
Q&A with Dr. Rabi Tawil, Part 2
This is a continuation of our Q&A session with Rabi Tawil, MD, co-director of the MDA Neuromuscular Disease Clinic at the University of Rochester, New York, and lead author of… Read More »
Antisense technology muzzles DUX4
The cause of facioscapulohumeral muscular dystrophy (FSHD) is thought to center on DUX4, a gene that normally is silent in adult skeletal muscle. When DUX4 gets “expressed,” as happens in… Read More »