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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June 28, 2024

Capricor Therapeutics Announces Positive Skeletal Muscle and Cardiac Function Data from HOPE-2 OLE Study Data of Deramiocel (CAP-1002)

Capricor Therapeutics today announced additional positive 3-year safety and efficacy results from the company’s ongoing HOPE-2 open label extension (OLE) study of deramiocel (CAP-1002) for the treatment of Duchenne. CAP-1002 is the company’s novel cell…

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June 24, 2024

REGENXBIO Announces Expansion of AFFINITY DUCHENNE® Trial to Include New Cohort of Younger Patients

REGENXBIO has announced it initiated enrollment in a new cohort of patients ages 1-3 in its Phase I/II AFFINITY DUCHENNE® trial. The trial is evaluating the safety and efficacy of RGX-202, an investigational gene therapy…

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June 12, 2024

Pfizer Shares Update on Phase 3 Study of Investigational Mini-Dystrophin Gene Therapy

PPMD is deeply disappointed to share that Pfizer Inc. announced today that CIFFREO, a Phase 3 randomized, double-blind, placebo-controlled study evaluating the company’s investigational mini-dystrophin gene therapy, fordadistrogene movaparvovec, in ambulatory patients with Duchenne did…

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