welcome to DuchenneXchange
- a positively charged Duchenne muscular dystrophy community.- join today!
- login
Hope in Translation: Bringing Gene Therapy to the Clinic
In 1987, scientists first uncovered the gene that is the target of mutations that causes Duchenne muscular dystrophy (DMD), a lethal muscle-wasting disease that affects about one in every 5,000 newborn boys. While the discovery of the dystrophin gene sparked hope that scientists could someday replace the mutated gene with a healthy one, turning this concept into a safe and effective treatment has been a decades-long challenge.
In drug discovery, the process of converting early biological insights into a therapy that can be tested in the clinic is known as translational research. It’s rigorous and very often painstaking work that happens behind the scenes, but the nonetheless is a critical bridge to bringing life-transforming treatments to patients. And in the journey of developing a gene therapy for DMD, scientists have had to overcome significant hurdles due to the complexity of the disease and the various challenges of engineering vector delivery systems.
rareRelated
-
UCLA research could be first step toward healing the hearts of children with DuchenneAfter a progressive weakening of the mus...
-
New lab technology could reveal treatments for muscle-wasting diseaseResearchers from Queen Mary University o...
-
U.S. appeals court upholds MIT, Harvard patents on CRISPR gene editingA U.S. appeals court on Monday allowed a...
-
Microdystrophin Gene Transfer Study in Adolescents and Children With DMDThis is a randomized, controlled, open-l...
-
A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy (DMD)The purpose of this study is to evaluate...
-
Sarepta Announces Clinical Hold Lifted for its Duchenne Muscular Dystrophy Micro-dystrophin Gene Therapy ProgramSarepta Therapeutics, Inc., a commercial...
-
Pfizer doses first patient using investigational mini-dystrophin gene therapy for the treatment of Duchenne muscular...Pfizer Inc. has initiated a Phase 1b cli...
