Researchers at the University of Texas Southwestern Medical Center in Dallas announced that they have made a significant advance in demonstrating the possibility of using CRISPR gene editing technology as a therapy for Duchenne muscular dystrophy (DMD). The technology was designed to “patch” a mutation in the dystrophin gene, which is defective in DMD, and… Read More »
Gene editing takes big step forward in Duchenne dog model
Facio broadens portfolio of potential drug development candidates
Reposted from Facio Therapies, Leiden, The Netherlands – August 30, 2018 Facio Therapies announced today that it has selected a second series of potential candidates for FSHD drug development. This series encompasses a family of novel small-molecule compounds that repress DUX4 by engaging a molecular target that differs from the target of Facio’s first series… Read More »
Why I made the FSH Society a beneficiary in my will
Because if not us, then who? by Deborah Schwartz, New York City I was clinically diagnosed with FSHD two years before the FSH Society came into being. Mine is a spontaneous mutation. I always had winged shoulder blades and rounded shoulders, and had started falling. As I knew no one who had this and there was… Read More »
Walk & Roll to Cure FSHD is coming next month!
by Beth Johnston and Leigh Reynolds, FSH Society Our first nationally branded event, the Walk & Roll to Cure FSHD, will take place in September and October 2018. This new, signature fundraising event is happening in five locations around the U.S., with additional sites being added in 2019. Find them here. Our five brave Walk &… Read More »
An update on early-onset FSHD
A review of published studies to improve our understanding by Amanda Hill, Highlands Ranch, Colorado As many as 20 percent of patients with FSHD have an “infantile” or “early-onset” form, which is generally understood to be more severe and more quickly progressing than typical FSHD. Historically, the criteria that have been used to define early-onset FSHD… Read More »