Growing numbers of companies and academic laboratories are pressing forward with early-stage drug development efforts. This chart shows how far various candidate anti-DUX4 drugs have progressed along the path to FDA approval. DUX4 is considered a key gene causing FSHD. The process begins with finding a “target,” or biological disease mechanism, that can be modified… Read More »
FSHD drug development pipeline
Get a MOVE on!
by June Kinoshita, FSHD Society When doctors diagnose individuals with a progressive condition like FSH muscular dystrophy (FSHD), they’re often asked, What does the future look like? Will I need to use an orthotic brace? How long do I have before I need to use a walker or a wheelchair? What about respiratory support? While… Read More »
FSHD Society releases Voice of the Patient Report
Landmark report captures compelling testimony by patients and family members about the severity of disease symptoms and urgent need for treatment LEXINGTON, MASS. (PRWEB) NOVEMBER 12, 2020 The FSHD Society has released its Voice of the Patient Report, a landmark publication based on its June 29, 2020, externally led patient-focused drug development (EL-PFDD) meeting. During the meeting,… Read More »
The remarkable origins of FSHD research in America
by Allison Calder, Salt Lake City, Utah Did you know the first grant ever paid out by the United States National Institutes of Health (NIH) was awarded to study FSHD? Maybe you are as shocked as I was when I first heard this while sitting in a family conference at the University of Utah a… Read More »
Feeling the urgency of NOW. Time = Lives
Last year, the FSHD Society launched an aggressive initiative to accelerate therapeutic development in FSHD. Because of our donors’ commitment and investment, we hosted the first Industry Collaborative Workshop for Therapy Development in FSHD with pharmaceutical companies, researchers, and regulatory and governmental agencies to identify the major obstacles to clinical trial development and regulatory approval… Read More »