In this FSHD University Webinar from April 15, 2021, Angela Lek, PhD, a research scientist at Yale University School of Medicine, speaks about how her husband’s diagnosis with limb-girdle… Read More »
A researcher’s journey into FSHD
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… 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… Read More »
What a difference 30 years have made
Time flies when you have your nose to the grindstone, working with focus and determination on a lofty goal. It has been an amazing 30 years since Daniel Perez and… Read More »
New biotech is developing Genea Biocells’ FSHD drug
By Alex Kiselyov, CEO, Myocea, Inc. Myocea is a biotechnology startup with a focus on neuromuscular disorders. Our product pipeline is championed by the small molecule GBC0905 (Rebastinib) aimed at… Read More »