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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September 24, 2021

Tomorrow is Coach To Cure MD 2021!

We are just one day away from the 14th Annual Coach To Cure MD presented by Werner Ladder on Saturday, September 25th! This weekend, AFCA Coaches around the country will be wearing Coach To Cure…

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September 24, 2021

Capricor Therapeutics Announces Positive Final Data From its Phase II HOPE-2 Trial in Patients with Duchenne Muscular Dystrophy Treated with CAP-1002

PPMD is pleased to share that Capricor Therapeutics announced today positive final data from the HOPE-2 randomized, double-blind, placebo controlled clinical trial using CAP-1002 to treat patients with Duchenne. The HOPE-2 clinical trial met its…

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September 22, 2021

PPMD Continues to Support Carrier-Focused Research with Latest Award

Throughout the years, I have learned a great deal from working with boys and young men with Duchenne and Becker. I have also come to know their families well and a common concern for families…

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September 15, 2021

2021/2022 Flu Season: Stay Safe, Informed, and Healthy

With all of the attention on COVID-19, we don’t want to lose track of the need to protect against the flu.  The flu isn’t going away, and as  flu season approaches, we want to help…

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September 9, 2021

Thanks to your continued investments PPMD continues forward in all areas of support

This Duchenne Action Month, PPMD is recognizing the strength of our community as we work to ensure that people with Duchenne thrive in adulthood. Thanks to your continued investments, our loved ones are living longer,…

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August 27, 2021

FDA’s Full Approval of Pfizer-BioNTech’s COVID-19 Vaccine Comarnaty: What Does It Mean?

We were excited to learn of the news of the FDA’s full approval of Pfizer-BioNTech’s Covid-19 vaccine, now marketed under the name Comarnaty, on Monday August 23, 2021. This is the first fully approved Covid-19…

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August 26, 2021

PPMD Invests $350,480 in Myosana Therapeutics to Support Non-Viral Gene Therapy Development

Parent Project Muscular Dystrophy is excited to announce a $350,480 programmatic investment in Myosana Therapeutics, Inc. (Myosana) to support the company’s early-stage development of a non-viral gene therapy delivery platform aiming to slow skeletal muscle…

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