Blog

FDA grants approval via accelerated approval pathway to ELEVIDYS: First-ever gene therapy for individuals with Duchenne ages 4-5

Today is an incredible milestone for the entire Duchenne community. It is a first step. But a huge step forward. Today, the FDA has granted accelerated approval to ELEVIDYS (delandistrogene moxeparvovec-rokl), a micro-dystrophin gene therapy…

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Norton Children's Hospital CDCC

PPMD Announces Pediatric Certified Duchenne Care Center at Norton Children’s Hospital

Today Parent Project Muscular Dystrophy announced the expansion of its renowned Certified Duchenne Care Center (CDCC) Program with the certification of the clinic at Norton Children’s in Louisville, KY. This is an exciting step for…

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Parents meet with doctor

PPMD publishes results of study to understand Duchenne patients’ and caregivers’ perspectives on corticosteroids

Earlier this month, the publication, A Mixed-Method Study Exploring Patient-Experienced and Caregiver-Reported Benefits and Side Effects of Corticosteroid Use in Duchenne Muscular Dystrophy, was accepted into the Journal of Neuromuscular Diseases. Background PPMD has led…

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PPMD Announces Pediatric Certified Duchenne Care Center at Boston Children’s Hospital

Today Parent Project Muscular Dystrophy announced the expansion of our renowned Certified Duchenne Care Center (CDCC) Program with the certification of Boston Children’s Hospital. This is an exciting step for the CDCC Program as it…

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Female patient sits with doctor

Over 1,000 Free Carrier Tests Performed through PPMD’s Decode Duchenne Program!

We are thrilled to announce a new milestone achieved in the Decode Duchenne genetic testing program. We recently reached 1,000 carrier tests performed! This means that over 1,000 women have received free carrier testing through…

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Boy at school

New Duchenne School Resources

Even though school is officially out for summer, it’s never too early to start planning for next year. We are here to help navigate this Duchenne journey together and connect you with any additional resources…

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PPMD resubmits RUSP nomination

Update on PPMD’s newborn screening efforts

For the past decade, PPMD has been leading efforts for newborn screening for Duchenne. As part of that process, PPMD, with co-sponsor Muscular Dystrophy Association (MDA), submitted a nomination package to the Recommended Uniform Screening…

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PPMD Joins Duchenne Parent Project aps Endocrine & Osteoporosis International Professional Care Meeting

How do we prevent the first fracture? How does weight & puberty affect bone health? How do we understand and implement new therapies? All of these questions and more will be covered this week as…

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PPMD’s Certified Duchenne Care Center Program – 2022 Impact & Progress

Every single person living with Duchenne deserves the best care possible. It is important that families know who is in charge of delivering the care they need and understand what centers are providing optimal, standardized…

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WATCH: Connect With the Experts – Bone Health in Duchenne (Webinar Recording)

Connect With the Experts – Bone Health in Duchenne PPMD recently hosted a webinar led by expert endocrinologists Dr. Leanne Ward (University of Ottawa), Dr. Janet Crane (Johns Hopkins) and Nadia Merchant (Children’s National) to…

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