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 »
Arrowhead Pharmaceuticals announces FSHD drug candidate
Arrowhead Pharmaceuticals, Inc., a Pasadena, California, company, today announced ARO-DUX4 as Arrowhead’s first muscle targeted investigational RNAi therapeutic candidate. Using the company’s proprietary Targeted RNAi Molecule (TRIMTM) platform, ARO-DUX4 is designed to target the gene that encodes human double homeobox 4 (DUX4) protein as a potential treatment for patients with facioscapulohumeral muscular dystrophy (FSHD). Pending… Read More »
A trio of promising advances toward treating FSHD
Commentary by Emanuele Mocciaro, PhD, San Raffaele Scientific Institute, Milan, Italy Facioscapulohumeral muscular dystrophy (FSHD) is caused by the “anomalous” reactivation of the DUX4 gene. DUX4 has an important role at the beginning of embryo development and is subsequently turned off and kept silent throughout life. But in FSHD, aberrant expression of DUX4 in muscle… Read More »
Partners in Pain, Harbingers of Hope
by Haviva Ner-David, Kibbutz Hannaton, Israel It was a chance meeting in the valley that divides my Galilean kibbutz Hannaton from the Bedouin village of Bir al Maksur that planted the seed for the storyline of my debut novel, Hope Valley. I was out walking my dog when I saw Hussein, a shepherd who frequents… Read More »
Notes on spinal and scapular surgery
Excessive curvature of the lower spine, a condition called hyperlordosis, can be a particularly painful and troublesome symptom of FSH muscular dystrophy. We are often asked whether abdominal braces or surgery can help. To answer this question, Dr. Ray Huml has kindly given us permission to share an excerpt by Drs. R.K. Lark and E.W…. Read More »
Hot off the press – FSHD Advocate
Our new issue of the FSHD Advocate, the first of 2021, is now available online. For those of you who receive our flagship magazine by the postal service, it will be arriving over coming weeks. As the cover and stories inside emphasize, the FSHD Society’s most important role in working toward a treatment is in… Read More »