The U.S. Food and Drug Administration (FDA) has given Pfizer the go-ahead to open U.S. sites for the Phase 3 CIFFREO clinical trial, testing the experimental gene therapy PF-06939926 in boys with Duchenne muscular dystrophy (DMD). The FDA had placed a hold on Pfizer’s application to start the trial in…

Treatment with PF-06939926, an experimental gene therapy for Duchenne muscular dystrophy (DMD), was generally well-tolerated among ambulatory patients — boys able to walk — in a Phase 1B clinical trial. Data also hint at the efficacy of the gene therapy, being developed by Pfizer, with researchers noting that…

Muscular Dystrophy News Today brought you daily coverage of the latest scientific findings, treatment developments, and clinical trials related to muscular dystrophy (MD) throughout 2021. We look forward to reporting more news to patients, family members, and caregivers dealing with MD during 2022. Here are the top 10 most-read articles of…

A young man with Duchenne muscular dystrophy (DMD), who was participating in Pfizer’s Phase 1B open-label clinical trial evaluating its gene therapy candidate PF-06939926, has died. The patient has only been identified as a young man who was not able to walk. In a statement, Pfizer expressed…

Pfizer has announced a delay in opening U.S. sites for its Phase 3 clinical trial CIFFREO, evaluating the investigational gene therapy PF-06939926 in boys with Duchenne muscular dystrophy (DMD). The delay is caused by questions…

Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. The investigational gene therapy PF-06939926 continues to demonstrate an acceptable safety profile and the potential for…

With the recent approval of Amondys 45 (casimersen) by Sarepta Therapeutics, there are now four exon-skipping therapies available in the U.S. for eligible patients with Duchenne muscular dystrophy (DMD). The others are Exondys 51 (eteplirsen) and Vyondys 53 (golodirsen), also by Sarepta, and Nippon Shinyaku’s…

The first boy has been dosed in a Phase 3 trial testing Pfizer‘s gene therapy, PF-06939926, in treating Duchenne muscular dystrophy (DMD). “The initiation of our pivotal trial, which is the first Phase 3 DMD gene therapy program to begin enrolling eligible participants, is an important milestone for the…

Throughout 2020, Muscular Dystrophy News Today brought you daily coverage of important discoveries, treatment advancements, clinical trial findings, and other relevant events related to muscular dystrophy. As we look forward to bringing you more news this year, we present here the 10 most-read stories of 2020, along with a…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to PF-06939926, Pfizer’s investigational gene therapy for Duchenne muscular dystrophy (DMD). This designation is given to treatments that show considerable potential in addressing serious conditions for which available treatments fall short. It is…