We’re thrilled to announce that researchers at Nationwide Children’s Hospital have documented the first-ever creation of full-length dystrophin in a human as a response to gene therapy. CureDuchenne has contributed […]
Pfizer’s Global Clinical Lead for Rare Neurological Diseases, Beth Belluscio, MD-PhD, and Debra Miller, Founder and CEO of CureDuchenne, sat down to educate the Duchenne community on Pfizer’s CIFFREO Trial […]
Entrada Therapeutics announced encouraging new data on their exon 44 skipping program following their presentation at TIDES USA 2022. CureDuchenne provided funding to Entrada last year as part of their Series […]
Initiation of AFFINITY DUCHENNE™ trial of RGX-202 for the treatment of Duchenne will be delayed. This January, we shared the news that REGENXBIO received clearance from the U.S. FDA and would […]
Pfizer’s Letter to the Duchenne Community We are pleased to share the good news that several regulatory authorities have approved the re-start of our Phase 3 ambulatory trial (CIFFREO) for […]
Today, Solid Biosciences announced a change to its corporate strategy to better align resources with goals to bring SGT-001 and SGT-003 to patients. Key Takeaways: Enrollment in IGNITE DMD, its […]
We are pleased to share that Edgewise Therapeutics is sponsoring a natural history study in Becker muscular dystrophy, to better understand the course of disease and define endpoints to be […]
In December 2020, CureDuchenne Ventures announced funding support for PepGen, Inc. to advance their Duchenne exon-skipping program to the clinic for trial. PepGen’s EDO cell-penetrating peptide platform is designed to improve the delivery of […]
Solid Biosciences issued a press release providing new, positive 2-year data from their IGNITE DMD clinical study in advance of the 2022 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference. […]
