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 […]
Solid Biosciences announced a widely anticipated update on their INSPIRE DUCHENNE Phase 1/2 clinical trial of SGT-003, their investigational micro-dystrophin gene therapy for Duchenne muscular dystrophy. What this means for […]
The FDA is continuing its review of Ataluren (Translarna), the first potential therapy specifically for individuals with nonsense mutation Duchenne (nmDMD). There is currently no set decision date. The application […]
CureDuchenne was an early investor in Dyne Therapeutics in 2020, and congratulates the company for receiving Breakthrough Therapy Designation from the US FDA for DYNE-251, their exon 51-skipping agent for the treatment of […]
Breaking News: Sarepta to resume shipments of ELEVIDYS for ambulatory individuals with Duchenne 07/28/25 5:09 PM EDT We are pleased to report that the US FDA has recommended that Sarepta […]
CureDuchenne was an early investor in Avidity Biosciences in 2018, and congratulates the company for receiving Breakthrough Therapy Designation from the US FDA for delpacibart zotadirsen (abbreviated as del-zota), their exon 44-skipping agent […]
Precision BioSciences is developing a novel gene editing approach for individuals with dystrophin mutations in the “hot spot” region of exons 45-55, with the goal of submitting applications later this […]
