Pfizer PFE announced that a phase III study on its mini-dystrophin gene therapy fordadistrogene movaparvovec for treating Duchenne muscular dystrophy (“DMD”) failed to meet the primary ...
The death of a young boy with Duchenne muscular dystrophy (DMD) who was treated with Pfizer’s experimental gene therapy in a phase 2 trial has prompted the company to pause dosing in another study.
Pfizer's two closest rivals in the DMD gene therapy space – Sarepta and Solid Biosciences – have both had their AAV-based gene therapies for DMD temporarily placed on clinical hold in the past ...
For example, Duchenne muscular dystrophy presents early ... and productive communication between regulators and gene therapy researchers. Pfizer is leveraging its global regulatory expertise ...
The young boy was suffering from Duchenne muscular dystrophy (DMD), a ... that the patient received the gene therapy in early 2023, as per a report from Pfizer. "We do not yet have complete ...
Pfizer also seeks FDA approval for its experimental antibody, marstacimab, to treat hemophilia A and B. The company is also developing a gene therapy for Duchenne muscular dystrophy, a genetic ...
New Gene Therapy Approach Shows Promise for Duchenne Muscular Dystrophy July 24, 2024 — Researchers have made a significant breakthrough in developing a new gene therapy approach that restores ...
Pfizer will voluntarily pull its sickle cell ... Vertex, CRISPR score landmark FDA approval for sickle cell disease gene therapy Casgevy Oxbryta’s retreat comes after the recent FDA approvals ...