Wave Life Science Exon Skipping in Duchenne Wave Life Sciences: Update on 48-week Data from FORWARD-53 Exon Skipping Study: An update on Wave’s Phase 2 FORWARD-53 trial of WE-N531, Wave’s […]
Wave Life Sciences Exon-skipping Trial Wave Life Sciences has shared new data from its ongoing FORWARD-53 clinical trial, evaluating the investigational exon-skipping therapy WVE-N531 for individuals with Duchenne muscular dystrophy […]
Entrada trial skipping exon 45 CureDuchenne provided early funding to Entrada Therapeutics, who has announced that they received authorization in the United Kingdom (UK) to initiate a Phase 1/2 multiple […]
CureDuchenne provided early funding to Capricor Therapeutics, and we are pleased to share that the FDA has accepted its Biologics Licenses Application (BLA) seeking approval for Deramiocel to treat cardiomyopathy in […]
Avidity Biosciences, which received early funding from CureDuchenne, plans to submit for US FDA Accelerated Approval at the end of 2025 for Delpacibart zotadirsen (del-zota), an exon-skipping experimental therapeutic for […]
Australian company Percheron Therapeutics released topline 6-month data from its Phase 2 study of avicursen in non-ambulatory individuals with Duchenne. The trial did not meet its primary endpoint, and there were […]
CureDuchenne is delighted to share the positive updates from REGENXBIO on their Phase 1/2 open-label clinical trial of RGX-202, an AAV8-delivered microdystrophin with an extended C-terminal domain. RGX-202 demonstrated robust […]
Avidity Biosciences, which received early funding from CureDuchenne, announced positive initial data from their EXPLORE44 Trial for individuals with Duchenne amenable to skipping exon 44. Their experimental exon-skipping agent, AOC […]
