By Rachel Tompa / Fred Hutch News Service A new study has revealed more players in the pathway of facioscapulohumeral muscular dystrophy, or FSHD, the most common form of muscular… Read More »
Study uncovers new proteins involved in regulating FSHD-linked gene
UC Irvine recruiting volunteers for study
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 »
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 »