The first Phase 3 clinical trial of DMD gene therapy completed the first patient's medication

DMD is a rare genetic disease caused by mutations in the genes that encode dystrophin on the X chromosome.
deficiency or defect of antimyostrophy protein, which causes chronic damage to the muscles during contraction, inflammation attacks, and affects muscle regeneration.
, muscles are replaced by scar tissue or fat. The symptoms of muscle weakness in patients with
are evident at the age of 2-3, when muscle tissue and function are constantly lost, they are usually only able to move in wheelchairs at age 12, need assisted breathing at age 20, and die early from breathing or heart failure at age 30-40.
Pf-06939926, developed by Pfizer, is an intravenous gene therapy.
it will be controlled by human muscle-specific initiation "mini-dystrophin" genetically modified in adeno-related virus 9 (AAV9) vector.
AAV9 virus vector has the ability to target muscle tissue to deliver genetically modified organisms.
the study has been approved by the FDA as an orphan drug, a rare pediatric disease drug, and a fast-track drug.
1b study released last year showed that nine DMD patients between the ages of 6 and 12 were treated with PF-06939926.
After 12 months of treatment, patients showed significant and lasting and statistically significant improvements, including consistent levels of micro-antimyostrophy protein expression (using liquid chromatography mass spectrometography and immunofluorescence staining), and improvements in The NorthStar Outpatient Assessment Scale (NSAA), a validated indicator of muscle function.
"Our critical clinical trials are the first phase 3 DMD gene therapy program to recruit patients, an important milestone for the patient population.
approved treatments that currently change the progress of the disease are available for all genetic types of DMD patients.
Dr. Brenda Cooperstone, Pfizer's Chief Development Officer for Rare Diseases, said, "We believe that this gene therapy, if successful and approved in Phase 3 clinical trials, has the potential to significantly improve the trajectory of DMD disease progression, and we are working with global regulators to launch this project as soon as possible in other countries."
: This article is intended to introduce medical and health research, not treatment options recommended.
if you need guidance on treatment options, visit a regular hospital.
resources: Pfizer Doses First Participant in Phase 3 Study for Duchenne Muscular Dystrophy Researchal Gene Therapy. Retrieved January 7, 2021, from。