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Carrier Care: Driving Improvements & Breaking Down Barriers

“Are you a carrier?,” they write in the hushed tones of private groups on social media, attaching anxious face and blue heart emojis in our new lexicon of connection and permission to express. What they…

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PPMD’s 2022 Annual Conference: Knight Hacks Poster Contest

Thanks to the generosity of PTC Therapeutics and members of the community, PPMD has established Knight Hacks: Everyday Tips and Tricks for Families, in memory of Angela Knight. One of Angela Knight’s enduring legacies is…

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Race Athlete Spotlight: Sarah James

This year, Sarah James ran the United Airlines NYC Half Marathon as part of the Race to End Duchenne team. Meet Sarah Why did you choose to join the Race to End Duchenne team? My…

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WATCH: Carrier Cardiac Care (Webinar Recording)

PPMD hosted a webinar on March 22, 2022 to discuss cardiac care for female carriers of Duchenne and Becker. Panelists discussed the importance of carrier health, learnings from the PPMD-sponsored carrier study at Nationwide Children’s…

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Santhera and ReveraGen Start Rolling NDA Submission to the FDA for Vamorolone for the Treatment of Duchenne

Santhera and ReveraGen have announced the initiation of a rolling new drug application (NDA) submission to the U.S. Food and Drug Administration (FDA) for vamorolone for the treatment of Duchenne. This is the first step…

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NIH Hosts Working Group to Better Understand Immune Response in Gene Therapy Trials (Webinar Recording)

Gene therapy, as we have often discussed, is still in its early days; meaning there are lots of lessons to be learned and new challenges that arise to ensuring all patients can one day have…

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PPMD Announces Pediatric Certified Duchenne Care Center at Monroe Carell Jr. Children’s Hospital at Vanderbilt

Today Parent Project Muscular Dystrophy (PPMD) announced the expansion of our renowned Certified Duchenne Care Center Program (CDCCP) with the certification of the clinic at Monroe Carell Jr. Children’s Hospital at Vanderbilt in Nashville, Tennessee….

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PPMD’s Power of Us Summit Series: Caregiver agenda is now available!

The Power of Us: Caregiver Summit — Agenda Is Now Available! April 28-30, 2022 | Atlanta, GA PPMD is excited to unveil our full agenda for The Power of Us: Caregiver Summit which will take place April 28-30,…

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Santhera and ReveraGen Present Efficacy and Safety Data with Vamorolone in Duchenne

Santhera and ReveraGen have shared an update on the evidence of clinical efficacy and safety of vamorolone in participants with Duchenne, based on the positive outcome of the pivotal Phase 2b VISION-DMD study and additional Phase…

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The BENEFIT Act Gets Positive Response From Witness During Congressional Hearing

We are grateful to Representative Matsui for asking a question on the BENEFIT Act during today’s House Energy and Commerce Committee hearing entitled “The Future of Medicine: Legislation to Encourage Innovation and Improve Oversight.” This…

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