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 […]
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 […]
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 […]
CureDuchenne is pleased to share that PTC Therapeutics has announced that the FDA has accepted for review the New Drug Application (NDA) for Translarna (ataluren) for the treatment of Duchenne […]
Watch the video HERE Pfizer recently provided this video updating the results from their Phase 3 micro-dystrophin gene therapy trial, CIFFREO, which were presented on October 12, 2024 at the […]
CureDuchenne welcomes the recent initiative the Food and Drug Administration (FDA) has taken to improve efficiencies in drug development, manufacturing, and the review process for new drug applications that incorporate […]
