In recent years, a growing understanding of myotonic dystrophy type 1 (DM1)—an often fatal, devastating, rare neuromuscular disorder that causes muscle weakness and other life-limiting ...

Del-desiran is the first investigational treatment for DM1 to receive Orphan Drug designation in Japan. Del-desiran has also received Breakthrough Therapy, Orphan Drug and Fast Track designations ...

Highlighted by Genentech's recent US regulatory submission for trastuzumab–DM1, antibody–drug conjugation technology could be heading for the mainstream in anticancer drug development.

Investing.com — Avidity Biosciences, Inc. (NASDAQ: RNA ), une entreprise biopharmaceutique spécialisée dans les thérapies à base d’ARN, a annoncé aujourd’hui que le ministère japonais de la Santé, du ...