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 […]
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 […]
Satellos Biosciences has received Investigational New Drug (IND) clearance from the FDA, as well as other global regulatory agencies, to conduct a 3-month placebo-controlled Phase 2 study of SAT-3247 in […]
Wave Life Sciences has announced that they are initiating Part C of the FORWARD-53 trial—an open-label Phase 1b/2 study of up to 15 ambulatory individuals aged 4-10 years and amenable […]
We congratulate C-PATH (Critical Path Institute) on their success in getting the FDA to qualify a new biomarker– glutamate dehydrogenase (GLDH)– to monitor drug-induced liver injury in clinical trials. This […]
Letter to the community added November 17, 2025 The FDA has approved the new labeling information Sarepta provided for Elevidys, Sarepta’s gene therapy for Duchenne. This update comes after reports […]
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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 […]
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 […]
