by Larry Chase Those of us who live with FSHD must “pivot” our world constantly. Pivot—the buzz word for what we all are going through and continue to experience, just like when you have “FSHD days” when it seems like there is no relief. My wife, our dog Zip, and I frequent a popular dog… Read More »
Pivot
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 »