September 5, 2024

Myology Course Educates and Advances Next Generation of Neuromuscular Physicians and Scientists

PPMD is dedicated to fighting for every future as we strive to end Duchenne. Since 2011, PPMD has been a proud supporter of the Myology Course, presented by Nationwide Children’s Hospital (NCH) and The Ohio…

Learn More

September 4, 2024

Introducing PPMD’s Newborn Screening Action Center: A Hub for Advocacy and Education

We are thrilled to announce the launch of PPMD’s Newborn Screening Action Center, a powerful resource designed to both educate and empower you as we work to ensure that every baby, in every state, has…

Learn More

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…

Learn More

September 1, 2024

Raising Our Voices This Duchenne Action Month

PPMD is excited to join forces with fellow Duchenne-specific organizations from around the world to celebrate World Duchenne Awareness Day on September 7! This global initiative, spearheaded by the World Duchenne Organization, aims to raise…

Learn More

August 23, 2024

Duchenne Added to Massachusetts Newborn Screening Panel

PPMD is excited to announce another significant milestone: Massachusetts has officially approved the addition of Duchenne to the state’s newborn screening panel! The addition of Duchenne to the Commonwealth’s newborn screen panel was included as…

Learn More

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…

Learn More

August 13, 2024

Finding Support to Manage Behaviors in Duchenne and Becker

Research has shown that dystrophin is usually present in the brain as well as in the muscles. Scientists believe that when dystrophin is missing or partially functional, like in Duchenne and Becker, brain neurons may…

Learn More

August 12, 2024

PPMD Urges FDA to Conduct a Full Review of Ataluren (Translarna) Following Resubmission of NDA

PTC Therapeutics has shared that it has resubmitted its New Drug Application (NDA) to the FDA for review and potential approval of ataluren (Translarna). Ataluren is an oral small molecule treatment that allows for stop-codon…

Learn More

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…

Learn More

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…

Learn More