This Q&A is based on the December 9 webinar with Rabi Tawil, MD, and Ghinwa Dumyati, MD, from the University of Rochester Medical Center, and the January 15 webinar hosted by the National Organization of Rare Diseases with Stephen Hahn, MD, FDA commissioner; Peter Marks, MD PhD, director of the FDA’s Center on Biologics Evaluation… Read More »
COVID-19 Vaccine Q&A
FSHD drug development pipeline
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 University Webinar – Fulcrum Clinical Trial Results
Join us on June 24 at 4:00 pm US ET for this special webinar on the findings from Fulcrum Therapeutics’ ReDUX4 clinical trial. The Fulcrum team will be sharing the data from its Phase 2b clinical trial of Losmapimod, a treatment for FSH muscular dystrophy that is intended to stop the expression of the DUX4… Read More »
The Job Talk
Lynn Stevens from Goodwill Industries of Northern Louisiana gave a superb webinar sharing much valuable information for anyone with a disability about resources to help people find and keep gainful employment. For anyone living with FSH muscular dystrophy, having this information handy can bring much peace of mind in planning the future. Noting that the… Read More »
Tell your doctor about the FSHD Masterclass
The FSHD Society is offering its first-ever CME-accredited masterclass on facioscapulohumeral muscular dystrophy (FSHD). This course, which will be held virtually on August 12, is of interest to any physician and allied health professional who sees adult and pediatric neuromuscular patients. They will be able to earn up to 4.25 continuing medical education (CME) credits—a… Read More »
Dave Younger’s exercise arsenal
by David Younger, Austin, Texas Over the past ten to fifteen years, I have developed an arsenal of products that I use to stay as healthy and active as possible. When COVID started, I bought two inexpensive lung exercisers on Amazon. I use the FIGERM breathing trainer twice a day, in the morning and in… Read More »
Dynamics of DUX4 downstream networks
by Lawrence J. Hayward, MD PhD, University of Massachusetts Medical School To develop robust therapies for FSHD, we need a better understanding of the sequence of events occurring at the single-cell level upon expression of DUX4, the causative gene. A recent paper by Chau et al. from the Yokomori lab at the University of California… Read More »