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…

Learn More

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…

Learn More

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…

Learn More

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…

Learn More

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…

Learn More

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…

Learn More

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…

Learn More

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…

Learn More

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…

Learn More

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…

Learn More