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Pfizer Shares Update on Phase 3 Study of Investigational Mini-Dystrophin Gene Therapy

PPMD is deeply disappointed to share that Pfizer Inc. announced today that CIFFREO, a Phase 3 randomized, double-blind, placebo-controlled study evaluating the company’s investigational mini-dystrophin gene therapy, fordadistrogene movaparvovec, in ambulatory patients with Duchenne did…

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Consensus Statement Featuring Expert Insights from PPMD’s Certified Duchenne Care Centers on Access to Novel Therapies for Duchenne Published

PPMD is excited to share that a consensus statement providing insights from clinicians who care for patients with Duchenne into the current therapeutic landscape and access to novel therapies for Duchenne has been published in…

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Roche Announces Phase II Open-Label SHIELD DMD Study to Assess Effect of Satralizumab on Bone Health in Duchenne

Today Roche announced plans for the SHIELD DMD study, a phase II open-label study to investigate the use of satralizumab in ambulatory and non-ambulatory individuals with Duchenne. Satralizumab is a monoclonal antibody that inhibits interleukin-6…

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Summer fun

Summer Fun: Accessible Activities for All Ages and Interests

As the school year wraps up, we are all looking forward to a fun-filled summer. It can be overwhelming to plan outings and activities considering accessibility and necessary accommodations to keep everyone safe. Along…

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Capricor Therapeutics Announces Positive 3-Year Efficacy Results from HOPE-2 Open Label Extension Study of CAP-1002

Capricor Therapeutics has announced positive 3-year results from the ongoing HOPE-2 open-label extension (OLE) study of CAP-1002 for the treatment of Duchenne. CAP-1002 is the company’s novel cell therapy. The HOPE-2 is a double-blind, randomized,…

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NS Pharma Shares Update on VILTEPSO® (Viltolarsen) Phase 3 Study

NS Pharma, Inc. has shared preliminary analysis results from RACER53, the global Phase 3 clinical trial of NS-065/NCNP-01 (viltolarsen). Viltolarsen is an antisense oligonucleotide drug intended to treat patients with Duchenne who are amenable to…

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Dyne Therapeutics Shares New Clinical Data from DELIVER Trial of DYNE-251 in Duchenne Patients

Dyne Therapeutics has shared new clinical data from the ongoing DELIVER trial of DYNE-251 in patients with Duchenne who are amenable to exon 51 skipping. DYNE-251 is an exon skipping product that combines a PMO…

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Study to Explore Barriers to Diverse Clinical Trial Participation in Duchenne Published

PPMD is pleased to share that the article, “Barriers to diverse clinical trial participation in Duchenne muscular dystrophy: Engaging Hispanic/Latina caregivers and health professionals,” has been published in the Orphanet Journal of Rare Diseases. This…

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PTC Therapeutics Announces European Commission Returns Translarna™ Opinion to CHMP For Re-evaluation

PTC Therapeutics, Inc. announced today that the European Commission (EC) has decided not to adopt the Committee for Medicinal Products for Human Use’s (CHMP) January 2024 negative opinion on the annual renewal of the conditional…

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Congress Passes FAA Reauthorization Featuring Monumental Accessibility Improvements

This week marked a significant milestone as Congress successfully passed the bipartisan Federal Aviation Administration (FAA) reauthorization, officially known as HR 3935, the Securing Growth and Robust Leadership in American Aviation Act. This legislation, now…

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