Sarepta Therapeutics announced positive topline data from the EMBARK trial, showing that Elevidys, 3 years post treatment, significantly slows disease progression on functional measures in ambulatory individuals who were 4-7 […]
Avidity Biosciences has released answers to frequently asked questions about their Managed Access Program (MAP) for individuals with Duchenne who are amenable to exon 44 skipping and meet clinical criteria. […]
World Duchenne Day 2025
A presentation with Dr. Doug Kerr, Chief Medical Officer at Dyne Therapeutics. Dr. Kerr shares topline results from the DELIVER clinical trial, along with new long-term data showing sustained benefits […]
As an early investor in Entrada Therapeutics, we are pleased to share their progress on their exon-skipping programs in Duchenne. For Exon 44: Entrada completed enrollment for the first cohort […]
Sarepta has completed the ESSENCE study, which is a confirmatory trial aimed at supporting full FDA approval of the exon-skipping PMOs Amondys 45 and Vyondys 53. The study did reinforce […]
Updated 10/31/2015:Letter to the community: October 30, 2025REGENXBIO announced that they have completed enrollment in the Phase 1/2 portion of their RGX-202 microdystrophin gene therapy trial, with topline data expected […]
Italfarmaco issues a letter to address the recent online discussions about the individuals who have passed away while being treated with Duvyzat (givinostat), confirming that they have determined the deaths […]
