January 7, 2021

Pfizer Doses First Participant in Phase 3 Study for Duchenne Investigational Gene Therapy

More exciting news for our community! Pfizer today announced that the first participant has been dosed in the Phase 3 CIFFREO study, which will evaluate the efficacy and safety of their investigational AAV9 mini-dystrophin gene…

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October 1, 2020

Pfizer Receives FDA Fast Track Designation for PF-06939926 Investigational Gene Therapy

Pfizer Inc. announced that its investigational gene therapy candidate (PF-06939926) being developed to treat Duchenne has received Fast Track designation from the FDA. PF-06939926 is currently being evaluated to determine the safety and efficacy of…

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September 21, 2020

Pfizer Shares Update on PF-06939926 Gene Therapy Trial During Company’s Virtual Investor Day

Pfizer recently shared an update on their mini-dystrophin replacement gene therapy product PF-06939926 during their virtual Investor Day. In total 18 boys have received the gene therapy product as part of the Phase 1b study….

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May 15, 2020

Pfizer Shares New Phase 1b Results of Gene Therapy in Ambulatory Boys With Duchenne

PROGRESS! Pfizer announced updated Phase 1b clinical data on PF-06939926, their investigational gene therapy being developed to treat Duchenne. The preliminary data from 9 ambulatory boys with Duchenne, aged 6 to 12 indicate that the…

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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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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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January 23, 2020

End Duchenne Tour Connect Group Session: Houston, TX

PPMD’s End Duchenne Tour made a stop in Houston, TX on Saturday, January 18. We were joined by over 40 people, including PPMD’s Connect Coordinators for South Texas, several PPMD families, and some of the…

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November 4, 2019

End Duchenne Tour Recap: Memphis, TN

PPMD’s End Duchenne Tour made a stop in Memphis, TN on Saturday, October 26. We were joined by over 60 people,  including families, an amazing group of volunteers in our kids room, six members of…

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September 24, 2019

End Duchenne Tour Recap: Wilmington, Delaware

PPMD’s End Duchenne Tour made a stop in Wilmington, Delaware on September 14. We were joined by over 100 people, including 30 families, 20 healthcare professionals and researchers, 15 industry partners, and 5 members of…

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