Recorded on January 20, this informational video discusses the upcoming Voice of the Patient Forum (or patient-focused drug development meeting) for facioscapulohumeral muscular dystrophy (FSHD) that is being organized by the FSHD Society for April 21. James Valentine explains the role of the patient’s voice in the FDA‘s consideration of drug approval applications. June Kinoshita… Read More »
Voice of the Patient Forum [VIDEO]
FAQs about genetic testing for FSHD
Julie Cohen, ScM, genetic counselor in the Center for Genetic Muscle Disorders at the Kennedy Krieger Institute in Baltimore, Maryland, answers some commonly asked questions. Q. What is the genetic cause of FSHD? Let me begin by saying that the genetics of FSHD is quite complex! There are two types of FSHD, called Type 1… 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 »
We Are Not Alone
BY DAVE LUKAS CRYSTAL LAKE, ILLINOIS The inaugural Chicagoland Walk & Roll to Cure FSHD exceeded every expectation and goal I had. What an amazing day filled with family, community, music, great food, fun, and picture-perfect weather! We had 230 walkers (my goal was 150). We raised $43,301 (my goal was $25,000). But more than… Read More »