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 dystrophy. Led by Fred Hutchinson Cancer Research Center biologist Dr. Stephen Tapscott and staff scientist Dr. Amy Campbell, the study, published today in the journal eLife, is the… 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 of California Irvine Department of Physical Medicine and Rehabilitation. The purpose of the study is to investigate a new method to measure functional mobility in… Read More »
Our new FSHD 101 video
Our new video explains the basic facts about facioscapulohumeral muscular dystrophy–the symptoms, how it affects those who have it, how it is inherited, and how many people are affected. Watch it and share it to help educate others about FSH muscular dystrophy. Thank you to Taylor Toole for compiling this video. Image and video credits:… 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 why it’s critically important to ramp up your support for our FSHD Therapeutics initiative, AND enroll in key studies such as the Clinical Trial Readiness study… 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 United States Food and Drug Administration (FDA) has granted orphan drug designation for ACE-083, the Company’s locally acting “Myostatin+” muscle agent, for the treatment of… Read More »