Treatments

Sarepta Therapeutics Announces FDA Approval of VYONDYS 53™ (golodirsen) Injection for the Treatment of Duchenne Muscular Dystrophy (DMD) in Patients Amenable to Skipping Exon 53 Source: Sarepta [...]

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 [...]

PTC Therapeutics announces new real-world analysis demonstrating Translarna™ (ataluren) slows disease progression in patients with Duchenne muscular dystrophy   La Force is happy to share [...]

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 [...]

PTC Provides Update on Translarna™ (ataluren) Application for Label Expansion June 28, 2019 > Original press release < PTC Therapeutics, Inc. announced that the Committee for Medicinal [...]

27 global sites currently open for enrollment Clinical trial sites in Europe are now open for enrollment in Bristol and Manchester, England; Munich and Hamburg, Germany; Dublin, Ireland; and [...]

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 [...]

Catabasis is enrolling boys ages 4 to 7 (up to 8th birthday), any mutation type, who have not been on steroids for at least the past six months.   What is Edasalonexent? Edasalonexent [...]