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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August 20, 2024

WATCH: Advancing DYNE-251 – An Investigational Medicine for Duchenne to Deliver for Patients (Webinar Recording)

Dyne Therapeutics recently joined PPMD for a presentation of clinical data from the DELIVER Trial of DYNE-251 in individuals living with Duchenne muscular dystrophy who are amenable to exon 51 skipping, first released on May…

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August 9, 2024

WATCH: Edgewise Therapeutics – Clinical Trials in Becker Muscular Dystrophy (Webinar)

Edgewise Therapeutics recently joined PPMD for a webinar to provide the Becker community information about the company’s clinical trials for Becker. Edgewise’s Chief Scientific Officer and Chief Medical Officer, shared two year results from the…

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August 9, 2024

Avidity Biosciences Announces Positive Data From Phase 1/2 EXPLORE44™ Trial of AOC 1044 in Individuals Amenable to Exon 44 Skipping

Avidity Biosciences, Inc. today announced positive data from the company’s Phase 1/2 EXPLORE44™ clinical trial of AOC 1044, an exon skipping therapy targeting those amenable to exon 44 skipping. Avidity also announced the approved international…

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August 6, 2024

Clinical Trials & Outcome Measures in Duchenne

Clinical trials and research studies are vitally important to improving health and quality of life for people with Duchenne and Becker. Research is always advancing and evolving, and outcome measures fall into this category, as…

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August 1, 2024

REGENXBIO Announces Positive Data From AFFINITY DUCHENNE® Trial of RGX-202

REGENXBIO Inc. has shared new, positive interim safety and efficacy data in the Phase I/II AFFINITY DUCHENNE® trial of RGX-202 in participants with Duchenne ages 1 to 11 years old. RGX-202 utilizes a novel adeno-associated…

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July 31, 2024

PepGen Announces Positive Data from Phase 2 Clinical Trial of PGN-EDO51

PepGen Inc. has shared positive clinical data from the first dose cohort (5 mg/kg) of PGN-EDO51, the company’s lead investigational candidate for patients with Duchenne whose mutations are amenable to exon 51-skipping. PGN-EDO51 is an…

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July 30, 2024

Pfizer Discontinues Development of Investigational Mini-Dystrophin Gene Therapy

PPMD is disappointed to learn that Pfizer Inc. has officially discontinued development of the company’s investigational mini-dystrophin gene therapy, fordadistrogene movaparvovec. This announcement follows Pfizer’s June 2024 update on the Phase 3 CIFFREO study evaluating…

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