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 13, 2022

Sign up today! Virtual Race Series-Power of Us

Sign up today for the Race to End Duchenne Virtual Race Series-May 21, 2022 – June 19, 2022! The Power of Us is stronger than any one of us alone. Join us for the Race…

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May 11, 2022

PPMD Announces 30th Certified Duchenne Care Center at Helen DeVos Children’s Hospital

Today Parent Project Muscular Dystrophy announced the expansion of our renowned Certified Duchenne Care Center (CDCC) Program with pediatric CDCC certification of the clinic at Spectrum Health Helen DeVos Children’s Hospital (HDVCH) in Grand Rapids,…

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May 5, 2022

Race Athlete Spotlight: Catalina Rodriguez

Catalina Rodriguez joined the Race to End Duchenne team and ran the runDisney Princess 5K in February of 2022.  With the support of her son and daughter, she finished with pride. Meet Catalina Why did…

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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 29, 2022

PPMD Brings Caregivers Together to Kick off The Power of Us Summit Series

PPMD Brings Caregivers Together to Kick off The Power of Us Summit Series Today PPMD is excited to convene the first meeting in The Power of Us Summit Series, our series of mental wellness retreats…

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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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