August 20, 2021

COVID-19 Vaccination Update

PPMD is closely following any changes or updates to current Covid-19 vaccine recommendations, including new recommendations for immunocompromised individuals. Under guidance of the Centers for Disease Control and Prevention (CDC) to understand how current guidelines…

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August 19, 2021

Race to End Duchenne in the most magical place on Earth!

Race to End Duchenne is offering THREE magical opportunities to join us this race season at Walt Disney World®! Disney’s Wine & Dine Half Marathon Weekend powered by AfterShokz (November 4-7, 2021) – This wickedly…

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August 18, 2021

PPMD’s Back-To-School Resources

PPMD’s Back-To-School Resources As summer comes to an end, PPMD is here to guide you through planning for a safe and successful school year. We have a collection of classroom resources on our website and…

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August 17, 2021

Solid provides updates on IGNITE DMD and SGT-003, next generation gene therapy pipeline initiative

Solid Biosciences has shared an update with the community on dosing as part of the IGNITE DMD Phase I/II clinical trial, developments for next generation gene therapy pipeline initiative SGT-003 and their collaboration with Ultragenyx….

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August 16, 2021

Edgewise Receives Fast Track Designation for EDG-5506 in Patients with Becker muscular dystrophy

Edgewise Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for rare muscle disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for…

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August 12, 2021

NS Pharma Shares Community Letter

August 12, 2020, marked the approval of Viltepso, the 4th FDA approved therapy for Duchenne. NS Pharma has posted the following community letter marking the occasion. Today we recognize the one-year anniversary of this approval….

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August 3, 2021

New Resource! Duchenne Carriers: Your Questions Answered

Duchenne Carriers: Your Questions Answered Being a carrier of Duchenne can affect so many aspects of a woman’s life, including health care, family planning, and mental health. As part of our efforts to better understand…

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July 29, 2021

Are You Ready for Kickoff? Coach To Cure MD Returns on Saturday, September 25th

Mark your calendars — Coach To Cure MD, presented by Werner Ladder, is back in-person and in a big way for its 14th season on Saturday, September 25th! Join our team and help #TackleDuchenne. Whether…

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July 22, 2021

PPMD Begins Process to Update Community-Led Duchenne Guidance for FDA

PPMD takes every action necessary to ensure therapies are developed efficiently and that they include the voice of the community. In 2014, we came together as a community, creating and submitting to the FDA the…

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July 20, 2021

Action Alert: Urge your House and Senate Members to Cosponsor the BENEFIT Act

We need your help! Please encourage your Senators to cosponsor the BENEFIT Act. This legislation will amend the Food, Drug and Cosmetic Act (FDCA) to ensure that patient experience, PFDD and related data – including…

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