Blog

PPMD Applauds FDA Launch of Rare Disease Innovation Hub — A Milestone for Rare Disease Patients

Parent Project Muscular Dystrophy (PPMD) welcomes the recent announcement by the United States Food and Drug Administration (FDA) of the “Rare Disease Innovation Hub”. This initiative marks a pivotal moment in the journey towards better…

Learn More

Capricor Therapeutics Announces Positive Skeletal Muscle and Cardiac Function Data from HOPE-2 OLE Study Data of Deramiocel (CAP-1002)

Capricor Therapeutics today announced additional positive 3-year safety and efficacy results from the company’s ongoing HOPE-2 open label extension (OLE) study of deramiocel (CAP-1002) for the treatment of Duchenne. CAP-1002 is the company’s novel cell…

Learn More

REGENXBIO Announces Expansion of AFFINITY DUCHENNE® Trial to Include New Cohort of Younger Patients

REGENXBIO has announced it initiated enrollment in a new cohort of patients ages 1-3 in its Phase I/II AFFINITY DUCHENNE® trial. The trial is evaluating the safety and efficacy of RGX-202, an investigational gene therapy…

Learn More

FDA Expands ELEVIDYS Label Indication for Ages 4 Years and Older

PPMD is excited to share that the FDA has made the decision to expand the labeled indication for ELEVIDYS, a micro-dystrophin gene therapy used to treat people with Duchenne muscular dystrophy, to include individuals at…

Learn More

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…

Learn More

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…

Learn More

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,…

Learn More

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…

Learn More

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…

Learn More

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…

Learn More

Join Our Mailing List