Gene therapy represents the vanguard for treating the world’s most deadly genetic diseases. Duchenne Muscular Dystrophy (DMD) is among a growing list of rare diseases where gene therapies are either approved for use by agencies such as the FDA and EMA or making their way through the late-stage clinical pipeline. Rare Disease Advisor’s DMD gene therapy resource center offers a range of news, insights, and resources on the research and development of DMD gene therapies.
Be sure to check back regularly as RDA continues to cover gene therapy news for the treatment of DMD.
Research on gene therapy has become increasingly prominent in recent years, and the trajectory is expected to continue. Since the Human Genome Project was completed a few decades ago, the collective imagination of scientists and physicians worldwide has been ignited by the possibilities that gene therapy opens up. Gene therapy offers the tantalizing prospect of…
A few decades ago, the prospect of gene therapy seemed to belong in the realm of science fiction. Now, gene therapy has come of age, and is in the process of becoming a serious form of therapy for use in many diseases, including Duchenne muscular dystrophy (DMD). "Gene therapy." In the minds of laypeople, this…
Jerry Mendell, MD, FAAN, Nationwide Children’s Hospital & Ohio State University, Columbus, OH, comments on the use of gene therapy in patients with Duchenne muscular dystrophy (DMD).
- Condition: Muscular Dystrophy, DuchenneIntervention: Genetic: PF-06939926Sponsor: PfizerNot yet recruiting
- A Phase 3 Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular DystrophyCondition: Duchenne Muscular DystrophyInterventions: Genetic: PF-06939926; Other: PlaceboSponsor: PfizerRecruiting
- A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular DystrophyCondition: Duchenne Muscular DystrophyIntervention: Genetic: PF-06939926Sponsor: PfizerActive, not recruiting
Read about the latest experimental therapies for DMD
Dr. Eric Olson Discusses the Use of Gene Therapy in the Treatment of DMD
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Eric Olson, PhD, chief scientific advisor at Vertex Pharmaceuticals and founding chair of the molecular biology department at the University of Texas-Southwestern Medical Center. Dr. Olson is an expert on Duchenne muscular dystrophy and CRISPR gene editing technology.