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
This webinar discusses a groundbreaking gene therapy treatment for Duchenne Muscular Dystrophy (DMD), focusing on a method to introduce a miniaturized version of the dystrophin gene using a viral vector. […]
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– ENTR-601-44 was well-tolerated in healthy volunteers with no serious adverse events, no drug-related adverse events and no clinically significant changes or trends noted in vital signs, ECGs, physical exams or […]
