gene therapy

Pfizer Shares Update on CIFFREO Phase 3 Gene Therapy Trial

Today Pfizer shared an update regarding their Phase 3 gene therapy trial, CIFFREO, with the community. While Pfizer has not yet opened sites within the U.S., the study is underway at 14 trial sites across…

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Webinar Q&A: Gene Therapy – Turning Obstacles into Possibilities

Last week, PPMD held a webinar to discuss the lessons learned in Gene Therapy, current challenges for our community, and what the future holds for these promising treatments. After opening with an insightful presentation from…

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PPMD Awards $31,500 Grant to Understand Immune Response to CRISPR/Cas-9 Gene Editing Strategies

PPMD is excited to award the University of Missouri School of Medicine a bridge grant for $31,500 to continue evaluating CRISPR therapy in a pre-clinical model of Duchenne. The project is led by Dongsheng Duan,…

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WATCH: Solid Biosciences Presents Safety & Efficacy Update (Webinar Recording)

On March 16, 2021, Solid Biosciences joined PPMD for a webinar to provide a safety and efficacy update on the ongoing IGNITE DMD phase I/II clinical study of SGT-001 microdystrophin gene therapy in patients with…

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Solid Reports Efficacy & Safety Data from Ongoing IGNITE DMD Clinical Trial & Resumption of Patient Dosing

Solid Biosciences has provided a safety and efficacy update on the ongoing IGNITE DMD phase I/II clinical study of SGT-001 microdystrophin gene therapy in patients with Duchenne. Upcoming Webinar: Join PPMD and Solid tomorrow, March…

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PPMD Awards University of Florida Wellstone Center $348,891 Grant for Duchenne Gene Therapy Research

PPMD is pleased to award the University of Florida (UF) Wellstone Muscular Dystrophy Cooperative Center a grant for $348,891. The project titled “Understanding and Improving AAV Gene Therapy for DMD” is led by H. Lee…

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February 17, 2021

Duchenne UK and PPMD Award $350,000 to Address Immunological Challenges of Gene Therapy in Duchenne

Parent Project Muscular Dystrophy (PPMD) and Duchenne UK are excited to announce Professor Kanneboyina Nagaraju at Binghamton, the State University of New York, as the recipient of our Joint Research Grant Call of 2020. The…

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Gene Therapy & Immune Response

February 10, 2021

Gene Therapy and Immune Response: Understanding Challenges and Strategies

PPMD recently announced the formation of the Pediatric Gene Therapy and Medical Ethics (PGTME) Working Group to address the challenges involved in gene therapy. As part of their recent educational series, the PGTME held a…

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January 21, 2021

Postponed: SRP-9001 Update – Community Webinar

Postponed: SRP-9001 Update UPDATE: Sarepta will have to postpone the community webinar scheduled for today, January 21 at 12 PM EST. PPMD is deeply disappointed as we were hopeful to discuss current data and ask…

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January 12, 2021

WATCH: PGTME Hosts Gene Therapy Lecture Series

As the Duchenne community enters the era of gene therapy, many new concepts, terms and issues arise, including those related to research design, patient autonomy, sibling and family involvement, fairness in access and the quality…

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