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 […]
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 […]
Study Co-Funded by CureDuchenne and cTAP Highlights Need for More Specific Enrollment Criteria That Could Increase Patient Participation and Drive More Comprehensive Therapeutic Evaluation CAMBRIDGE, Mass., July 24, 2024 – […]
We at CureDuchenne are excited to acknowledge the recent announcement by the United States Food and Drug Administration (FDA) about the creation of the “Rare Disease Innovation Hub.” This new […]
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CureDuchenne Webinar | ITF THERAPEUTICS Patient Community Update: An overview of DUVYZAT Recorded on June 26, 2024Watch HERE
