Solid Biosciences today reported an update on the progress of the INSPIRE DUCHENNE open label, Phase I/II clinical trial with SGT-003 and their planned regulatory discussions in 2026. 23 Pediatric […]
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 […]
Updated: December 4, 2025 From Belief to Breakthrough: Celebrating Avidity’s Potential Acquisition by Novartis In 2018, CureDuchenne made a bold investment in a promising company with an idea that could […]
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 […]
Capricor Therapeutics, which received early funding from CureDuchenne, provided an update on their Deramiocel program in Duchenne after their recent FDA meeting. This meeting with the FDA was to address […]
As an early investor in Avidity Biosciences, we are pleased to share their exciting news that skipping exon 44 in the dystrophin gene with Del-zota was associated with functional improvements […]
Results published in the Annals of Clinical and Translational Neurology show that long-term treatment with givinostat, a histone deacetylase (HDAC) inhibitor, in combination with corticosteroids, delayed disease progression in ambulant individuals with […]
In this webinar, Dr. Naz Dastgir, Executive Medical Director and Clinical Development Lead for RGX-202 at REGENXBIO, shared information on the active and enrolling AFFINITY DUCHENNE trial and how families […]
