May 18, 2022

Win a NEW Xbox Series X with PPMD’s Duchenne Registry!

Win a NEW Xbox Series X! For all of you gamers and parents of gamers, PPMD’s Duchenne Registry is excited to announce we will be giving away a brand new Xbox Series X to one…

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May 16, 2022

WATCH: PPMD & Pfizer Discuss Updates on CIFFERO Phase 3 Gene Therapy Trial

Recently Pat Furlong, PPMD’s founder and CEO, sat down with Dr. Beth Belluscio, Pfizer’s Global Clinical Lead of Rare Neurological Disorders, to discuss Pfizer’s learnings from previous gene therapy studies and updates on the CIFFERO…

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May 13, 2022

Entrada Therapeutics Shares New Data on ENTR-601-44 Exon Skipping Therapy

Entrada Therapeutics has shared new data from a preclinical study evaluating ENTR-601-44, an exon-skipping therapy set to target individuals with Duchenne amenable to exon 44 skipping. We are pleased to hear new data on this…

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May 4, 2022

Regenxbio Announces Delay in Timeline for Initiation of First Participant Dosed in Affinity Duchenne Trial

REGENXBIO today announced that it will delay the initiation of dosing in the Phase I/II AFFINITY DUCHENNETM trial studying RGX-202 in participants with Duchenne, due to an unexpected observation in the final stages of manufacturing…

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April 28, 2022

Pfizer Announces Clinical Hold Lifted for its Mini-Dystrophin Gene Therapy Program

Pfizer has issued a community letter sharing that the FDA has lifted the clinical hold on the company’s Phase 3 ambulatory trial (CIFFREO) for their investigational mini-dystrophin gene therapy product for Duchenne. Pfizer has addressed…

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April 27, 2022

Solid Biosciences Shares Update on Duchenne Gene Therapy Pipeline Programs

Today Solid Bio shared an update on their gene therapy programs for SGT-001 and SGT-003. SGT-001 is Solid’s AAV9 microdystrophin gene therapy investigational product that is currently being evaluated in boys with Duchenne. SGT-003 is…

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April 22, 2022

Astellas Gene Therapies Announces plans to discontinue development of Duchenne gene therapy programs

Last night, based on recent preclinical data, Astellas Gene Therapies announced that the research and development of gene therapy programs AT702, AT751 and AT753 for Duchenne Muscular Dystrophy have been terminated. • AT702 was designed…

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April 20, 2022

WATCH: Twice Weekly Steroids & Exercise for Duchenne – Study Update & Preliminary Findings (Webinar Recording)

Dr. Tanja Taivassalo joined PPMD for a community webinar last week to present an update on the University of Florida’s Department of Defense funded clinical trial evaluating the potential of low-dose twice weekly steroids and…

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April 15, 2022

Edgewise-Funded Natural History Trial Of Becker Muscular Dystrophy Now Enrolling

Edgewise Therapeutics has announced the start of an observational trial in participants with Becker muscular dystrophy to understand the disease progression as assessed by functional measures and imaging endpoints. The trial is expected to recruit…

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April 14, 2022

PPMD Announces First Patients Consented into EHR Study!

PPMD is excited to announce the launch of our new Electronic Health Record (EHR) Study, which will extract data from several of our Certified Duchenne Care Centers across the country, with the first patients now…

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