Research

The U.S. Food & Drug Administration (FDA) had accepted the filing of a New Drug Application (NDA) under the priority review for viltolarsen in patients with Duchenne Muscular Dystrophy (DMD) [...]

Correcting nonsense mutations with CRISPR / Cas9? Would it be possible to correct nonsense mutations with CRISPR/Cas9? Some patients with DMD have a point mutation (nonsense) that leads either to [...]

Santhera Announces Presentation by ReveraGen of Positive 18-Month Data with Vamorolone in Duchenne Muscular Dystrophy   La Force is happy to share this press release provided by Santhera [...]

Phase 3, PolarisDMD trial of edasalonexent in DMD has exceeded target enrollment   La Force is happy to share the latest edition of the Catabasis Connection newsletter   Catabasis [...]

Sarepta Therapeutics receives Complete Response Letter from the US Food and Drug Administration for golodirsen New Drug Application Sarepta Therapeutics, Inc announced it had received a Complete [...]

Catabasis announces that all 40 sites, across eight countries, are now fully launched for the Phase 3 PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy! La Force is happy to share [...]

Thanks to Bio Space, IOS Press, Science Daily for the content of this blog. Duchenne muscular dystrophy is characterized by progressive muscle degeneration. In DMD patients, the pulmonary [...]

Duchenne muscular dystrophy (DMD) is a disease that almost exclusively affects boys and whose incidence is 1 in 3,500 – 5,000. It is extremely rare that Duchenne muscular dystrophy (DMD) will [...]

FibroGen Receives Orphan Drug Designation from the U.S. FDA For Pamrevlumab for the Treatment of Duchenne Muscular Dystrophy   FibroGen, Inc. a leading biopharmaceutical company discovering [...]

Audentes Therapeutics, Inc., a leading Adeno-associated virus (AAV)* based genetic medicines company focused on developing and commercializing innovative products for serious rare neuromuscular [...]