“Never give up, for that is just the place and time that the tide will turn.”–Harriet Beecher Stowe As we enter a new decade – one that we believe will see effective therapies become available – the families that form the FSHD Society face new challenges. This year, we have an unprecedented opportunity to participate… Read More »
FSHD Advocate on the Road to Clinical Trials
Genetic testing for FSHD—a new frontier
There are exciting new developments in FSHD genetic testing. A new genetic test for FSHD is now being offered by PerkinElmer Genomics, a global company known for scientific and medical testing services. Other companies and groups are likely to follow suit. This is the first major innovation in FSHD genetic testing in nearly three decades… Read More »
Losmapimod receives orphan drug designation
Fulcrum Therapeutics has announced that losmapimod has received orphan drug designation from the U.S. Food and Drug Administration. Losmapimod is currently in a Phase 2 clinical trial for facioscapulohumeral muscular dystrophy (FSHD). The drug qualified for this status because FSHD, with an estimated 20-40,000 affected individuals in the U.S., is considered a rare disease. (The… Read More »
Fulcrum’s approach to treating FSH muscular dystrophy [VIDEO]
In this video, Michelle Mellion, MD, medical director at Fulcrum Therapeutics, discusses the Cambridge biotech’s approach to treating facioscapulohumeral muscular dystrophy. She describes the company’s scientific research to develop “FSHD in a dish” models to screen and identify candidate drugs that can suppress the expression of DUX4, the gene thought to be the trigger for… Read More »
FSHD goes to school
by Kristin Zwickau New England Chapter Director Having a child diagnosed with a complex, serious disease that causes disabilities is a very hard reality for parents to deal with, let alone for the child who faces it day in and day out. Our daughter, who was diagnosed at the age of 6, is mobile, but… Read More »