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 »
Arrowhead Pharmaceuticals announces FSHD drug candidate
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 »
MRI in FSH muscular dystrophy research
This month’s FSHD University webinar (video above) provided an outstanding, nontechnical explanation of magnetic resonance technology (MRI) and how it is being applied in the Fulcrum Therapeutics clinical trial… Read More »