March 27, 2020

Pfizer Shares Update on PF-06939926 Gene Therapy Trial and COVID-19

Pfizer has shared an update regarding the PF-06939926 gene therapy trial and COVID-19. Read Pfizer’s update: Our Clinical Trials UPDATE: March 25 —To avoid adding to the demands on the healthcare system during this unprecedented…

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March 19, 2020

Sarepta Therapeutics Shares Community Update on COVID-19

Sarepta Therapeutics has provided the community with an update addressing questions from the community regarding COVID-19, including updates on current clinical trials and accessing approved therapies. Read Sarepta’s Update March 19, 2020 Dear Duchenne and…

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March 12, 2020

Solid Biosciences Provides Letter to the Duchenne Community: Data and IGNITE DMD Clinical Trial Update

Solid Biosciences Inc. provided an update on the IGNITE DMD phase I/II microdystrophin gene therapy clinical program. Solid shares that biomarker data from all three patients dosed in the 2E14 vg/kg cohort of IGNITE DMD…

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February 28, 2020

Pfizer Shares Additional Information About PF-06939926 Gene Therapy Trial

Pfizer has provided additional information about their PF-06939926 gene therapy trial. PPMD will continue to share information on this trial as it becomes available. Read Pfizer’s community letter: On Monday, February 24th, 2020, the National…

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February 25, 2020

Pfizer Announces Recruitment Information for PF-06939926 Gene Therapy Trial

Yesterday the information for Pfizer’s Phase 3, randomized, double-blind, placebo-controlled study of PF-06939926, exploring the safety and efficacy of gene therapy in patients with Duchenne was posted to clinicaltrials.gov. This will be the largest cohort of patients…

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December 23, 2019

Sarepta Announces Partnership with Roche Outside the U.S. for its Investigational Micro-dystrophin Gene Therapy

Sarepta Therapeutics, Inc. today announced that Sarepta and Roche have entered into a licensing agreement providing Roche exclusive commercial rights to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin), Sarepta’s investigational gene therapy for Duchenne muscular dystrophy, outside the United States….

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December 20, 2019

Sarepta Therapeutics Shares Winter 2019 Newsletter

Sarepta has shared the Winter 2019 issue of their newsletter, including updates on current Sarepta programs. Click here to download.

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December 18, 2019

PPMD Awards $465,000 Grant to The Ohio State University to Rescue Cardiac Dystrophin & Function in Duchenne

Parent Project Muscular Dystrophy is excited to announce that Renzhi Han, an associate professor at The Ohio State University College of Medicine, will receive a $465,000 grant to study the potential use of in vivo…

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December 18, 2019

Solid Biosciences Provides Data Update from SGT-001 Development Program

Solid Biosciences Inc. provided an update on the IGNITE DMD phase I/II microdystrophin gene therapy clinical program. Solid shares that the status of the sixth patient dosed in the higher 2E/14 vg/kg, who experienced the…

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December 4, 2019

Astellas Enters into Definitive Agreement to Acquire Audentes Therapeutics

Astellas Pharma and Audentes Therapeutics announced that they have entered into a definitive agreement for Astellas to acquire Audentes. We look forward to learning more as this partnership moves forward. Read the Announcement from Audentes…

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