November 7, 2024

Solid Biosciences Shares Updates on INSPIRE DUCHENNE Clinical Trial of SGT-003

Yesterday, Solid Biosciences shared new updates on the INSPIRE DUCHENNE clinical trial for their next-generation AAV micro-dystrophin gene therapy candidate, SGT-003, for the treatment of Duchenne. Based on early study results, the trial has expanded…

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November 6, 2024

Sarepta Announces Discontinuation of SRP-5051 Development for Duchenne

Sarepta Therapeutics today announced that the company is discontinuing its SRP-5051 (vesleteplirsen) development program, including the global, Phase 2, multi-arm, ascending dose MOMENTUM study. Vesleteplirsen is an investigational, next-generation treatment utilizing Sarepta’s PPMO chemistry and…

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November 5, 2024

WATCH: Exploring Muscle Regeneration – A New Approach with Satellos (Webinar Recording)

Satellos recently joined PPMD for a community webinar exploring a new approach for the treatment of Duchenne. Satellos presented on the discovery behind SAT-3247, a small molecule drug that mobilizes the body’s own muscle stem…

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October 30, 2024

FDA Accepts Translarna NDA Resubmission for Review

A Letter From PPMD’s Pat Furlong I am thrilled to share that the FDA has officially accepted PTC Therapeutics’ New Drug Application (NDA) resubmission for Translarna (ataluren). This acceptance is an important step, meaning the…

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October 11, 2024

WATCH: SHIELD DMD – A Bone Health Study in Duchenne (Webinar Recording)

Bone health is an important aspect of living with Duchenne muscular dystrophy. Roche recently joined PPMD for a webinar during which Maitea Guridi, a Principal Clinical Scientist at Roche, shared learnings around the natural history…

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October 9, 2024

Capricor Initiates Rolling Submission of Biologics License Application (BLA) for Deramiocel (CAP-1002)

Capricor Therapeutics Initiates Rolling Submission of Biologics License Application (BLA) for Deramiocel (CAP-1002) for the Treatment of Duchenne Cardiomyopathy Capricor Therapeutics announced it has begun the rolling submission of its Biologics License Application (BLA) with…

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October 3, 2024

WATCH: Interim Results from FORWARD-53 Study of WVE-N531 in Duchenne Muscular Dystrophy (Webinar Recording)

Wave Life Sciences recently joined PPMD for a presentation of interim results from the company’s ongoing Phase 2 FORWARD-53 study of WVE-N531 in individuals living with Duchenne muscular dystrophy who are amenable to exon 53…

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September 24, 2024

Capricor Therapeutics Plans to File Biologics License Application for Full Approval of Deramiocel for the Treatment of Duchenne Cardiomyopathy

Capricor Therapeutics announced today its intent to file a Biologics License Application (BLA) seeking full approval of deramiocel for the treatment of Duchenne-cardiomyopathy based on existing cardiac data from their Phase 2 trials of deramiocel…

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September 24, 2024

Wave Life Sciences Announces Positive Interim Data from FORWARD-53 Clinical Trial Evaluating WVE-N531 in Individuals With Duchenne Amenable to Exon 53 Skipping

Wave Life Sciences has announced positive interim data from the ongoing Phase 2 FORWARD-53 study of WVE-N531, an exon skipping oligonucleotide being investigated for the treatment of Duchenne in those who are amenable to exon…

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September 3, 2024

Dyne Therapeutics Announces Positive New Clinical Data from Phase 1/2 DELIVER Trial of DYNE-251 in Duchenne

Dyne Therapeutics, Inc. has announced positive new clinical data from its ongoing Phase 1/2 DELIVER trial of DYNE-251 in patients with Duchenne who are amenable to exon 51 skipping. DYNE-251 is an exon skipping product…

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