October 7, 2021

Dyne Therapeutics Presents New Preclinical Data on the Potential of FORCE Platform to Address the Genetic Basis of Duchenne

Dyne Therapeutics presents new preclinical data on the potential of FORCE platform to address the genetic basis of Duchenne. Dyne Therapeutics recently released new preclinical data from their Duchenne muscular dystrophy program, which was initially…

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October 6, 2021

PPMD Completes Successful Pilot in Newborn Screening for Duchenne Muscular Dystrophy

PPMD’s Newborn Screening Pilot recently completed two years of ground-breaking recruitment. More than 36,000 families consented to participate and more than 36,000 babies have been screened for Duchenne Muscular Dystrophy in New York State. As…

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October 5, 2021

PPMD releases results of second survey on impact of pandemic on families

Since the start of the COVID-19 pandemic, Parent Project Muscular Dystrophy (PPMD) has surveyed Duchenne and Becker families to better understand how the patient experience with care, clinical trials, and access to approved therapies has…

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October 4, 2021

Sarepta Therapeutics in partnership with Roche announced the initiation of the EMBARK clinical trial for their investigational gene therapy product SRP-9001

Sarepta Therapeutics, in partnership with Roche, has announced the initiation of study SRP-9001-301, also known as EMBARK, a pivotal study of SRP-9001 (rAAVrh74.MHCK7. micro-dystrophin) for the treatment of Duchenne muscular dystrophy. It is important to…

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October 1, 2021

Duchenne Gene Therapy Update

As many in the community are aware, on Monday, September 28, Pfizer released a statement to the public announcing a change in their Phase III gene therapy trial for Duchenne. We applaud their efforts to…

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September 29, 2021

Wave Life Sciences Announces Initiation of Dosing in Phase 1b/2a Duchenne Clinical Trial of WVE-N531 Starting in Canada and the UK

  This morning, Wave Life Sciences Ltd. announced the initiation of dosing in a Phase 1b/2a clinical trial expecting to enroll 15 boys, evaluating investigational WVE-N531 as a treatment for boys with Duchenne muscular dystrophy…

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September 29, 2021

WATCH: Connect with the Experts – Cardiac Care

Watch: Connect with the Experts – Cardiac Care Last week PPMD’s Rachel Schrader moderated a webinar with expert cardiologists to discuss important topics related to cardiac medications, imaging, and advanced cardiac care. PPMD recognizes families…

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September 28, 2021

Pfizer Announces Amendment to Ongoing Gene Therapy Phase III Trial

Today Pfizer announced that they have submitted a protocol amendment to their ongoing Phase III trial of their gene therapy candidate (fordadistrogene movaparvovec).  Across Pfizer’s studies, three Severe Adverse Events (SAE) have occurred leading Pfizer…

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September 27, 2021

ReveraGen and Santhera Announce FDA Orphan Grant Funding for Clinical Trial with Vamorolone in Becker Muscular Dystrophy

PPMD is pleased to share that ReveraGen Biopharma and Santhera Pharmaceuticals announced that ReveraGen has received a $1.2 million grant from the FDA under their “Clinical Studies of Orphan Products Addressing Unmet Needs of Rare…

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September 27, 2021

Sarepta Therapeutics Plans to Initiate Part B of the MOMENTUM Study of SRP-5051 in Patients with Duchenne Following Positive Interactions with FDA

PPMD is pleased to share that Sarepta Therapeutics plans to initiate Part B of the MOMENTUM study of SRP-5051 in patients with Duchenne muscular dystrophy following positive interactions with the U.S. Food and Drug Administration…

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