Volunteers who are 18 to 55 years of age with FSHD are needed for a study to evaluate functional mobility. The principal investigator is Jay Han, MD, of the University… Read More »
UC Irvine recruiting volunteers for study
DUX4-suppressing therapies are nearing the clinic
From our 2018 FSHD Connect conference, researchers present their work on therapies to suppress the toxic DUX4 gene. Two companies may be going into clinical trials in 2019. This is… Read More »
Acceleron Receives FDA Orphan Drug Designation for ACE-083
From BusinessWire CAMBRIDGE, Mass.–(BUSINESS WIRE)–Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical company in the discovery and development of TGF-beta therapeutics to treat serious and rare diseases, today announced that the… Read More »
FDA orphan drug designation for Genea Biocells’ FSHD drug candidate
Genea Biocells, a San Diego-based biotech company focused on drug development to treat neuromuscular diseases, today announced that it has been granted Orphan Drug Designation by the U.S. Food and… Read More »
DUX may be a master switch of the genome
The DUX4 gene in FSH muscular dystrophy is typically described as a rogue actor, a genetic oddball that is never supposed to be active in adult muscle, and is rendered… Read More »