Capricor Therapeutics, which received early funding from CureDuchenne, said the FDA has requested the full clinical study report from their latest clinical trial, the HOPE-3 trial. Positive topline results from […]
REGENXBIO announced new, positive 18-month functional data from patients treated with the pivotal dose in the Phase I/II portion of the AFFINITY DUCHENNE® trial. They expect to share pivotal topline data […]
In 2018, CureDuchenne was the only Duchenne-focused organization to invest in Avidity Biosciences, recognizing the potential of their novel RNA-targeting approach for individuals with Duchenne muscular dystrophy long before it reached clinical trials. […]
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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 […]
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 […]
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 […]
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 is sharing a community letter from Sarepta announcing a strategic company restructuring and pipeline prioritization plan. Additionally, Sarepta shared that the U.S. FDA has requested and Sarepta has agreed to include a […]
