September 15, 2023

PTC Therapeutics Announces Negative European Medicines Agency Opinion Regarding Full Authorization of Translarna

PTC Therapeutics today shared that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended not renewing the Marketing Authorization for Translarna™ (ataluren), a treatment for Duchenne patients…

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August 30, 2023

FibroGen Announces Topline Data from Phase 3 LELANTOS-2 Study

FibroGen has announced topline results of the Phase 3 LELANTOS-2 trial of pamrevlumab, their investigational anti-fibrotic monoclonal antibody, for ambulatory patients with Duchenne muscular dystrophy.  Although generally safe and well tolerated, pamrevlumab did not meet…

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August 22, 2023

PPMD Awards $65,000 to Vanderbilt University Medical Center to Advance Understanding of Duchenne Cardiomyopathy

PPMD is excited to award Dr. Jonathan Soslow (Vanderbilt University Medical Center), and his collaborators Drs. Larry Markham (Riley Children’s Hospital) and Christopher Spurney (Children’s National Medical Center), a $65,000 grant to characterize microRNA and…

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August 2, 2023

PPMD Publishes 15 Year Registry Report 

PPMD is excited to announce the release of the 15 Year Registry Report. This report is the culmination of data provided by registrants in The Duchenne Registry, as well as collaborative work with expert clinicians…

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June 27, 2023

Edgewise Therapeutics announces positive topline 12-month data from EDG-5506 ARCH study

Edgewise Therapeutics today announced topline 12-month data from the ARCH study of EDG-5506, an orally administered small molecule designed to prevent contraction-induced muscle damage in patients with Becker muscular dystrophy. The ARCH study is evaluating…

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June 19, 2023

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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June 15, 2023

PPMD, CureDuchenne, Muscular Dystrophy Association Announce Collaborative Project to Focus on Re-Dosing Gene Therapy in Duchenne Muscular Dystrophy

CureDuchenne, Muscular Dystrophy Association (MDA), and Parent Project Muscular Dystrophy (PPMD) today announced a collaborative clinical trial grant to test repurposing of the FDA-approved drug Vyvgart (developed by argenx), for its potential ability to reduce…

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June 13, 2023

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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May 31, 2023

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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April 27, 2023

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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