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 ...
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 ...
Opens in a new tab or window SAVANNAH, Ga. -- Delandistrogene moxeparvovec (Elevidys), a gene therapy approved to treat Duchenne muscular dystrophy, missed its primary endpoint but differences ...
It is a type of gene therapy that aims to restore the production of dystrophin protein in the muscles, which can help slow down or stop the progression of DMD. WVE-N531 has shown strong results in ...