Positive results in the gene therapy micro-dystrophin trial to treat DMD

One of the objectives of our team is to inform you about new treatments. Here’s the latest news from Sarepta Therapeutics. They announce that at the 23rd International Congress of the World Muscle Society, Jerry Mendell, M.D., presented positive updated results from the four children dosed in the gene therapy micro-dystrophin trial to treat patients with Duchenne Muscular dystrophy. Enjoy reading and thank you for sharing these articles within the DMD community. -Press release from Sarepta Therapeutics: Sarepta news release.


A quick overview of gene therapy micro-dystrophin

This therapy uses a virus (the adeno-associated virus, or AAV) to deliver the micro-dystrophin, a shorter version of the dystrophin gene, which contains the minimum amount of information needed to produce a functional protein of dystrophin.* We are hopeful that, one day, micro-dystrophin will be a viable treatment for Duchenne muscular dystrophy (DMD).


What are the positive results?

  • Robust expression of micro-dystrophin
  • A significant decrease in creatine kinase (CK)
  • Improvements in all measured functions,
  • No serious adverse events (SAEs) were observed in the study.
  • Press release from Sarepta Therapeutics: Sarepta news release.


Words from Dr. Mendell

“The goal of this study was to validate what we observed in preclinical models. We observed efficient transduction of our vector, AAVrh74, to all muscle types; robust expression in skeletal muscles via the MHCK7 promoter; a reduction in creatine kinase levels; and a favorable safety profile. Similar to preclinical models, we also observed in this early study that robust expression has the potential to positively impact the natural course of disease progression.”


Words from Doug Ingram, Sarepta’s president and chief executive officer

“The encouraging results that we previously saw and reinforced in the fourth patient strengthen our resolve to rapidly move to a confirming trial and, assuming successful, to bring this therapy to the Duchenne community around the world with a sense of urgency.”


Words from Mr. Ingram

“These results create for us an obligation to patients around the globe living with and being damaged by this cruel disease. We are investing our energy, resources and creativity to moving the development forward, planning meetings with the FDA and other agencies around the world to take their input, building compelling access and reimbursement package, and establishing sufficient manufacturing capacity to fully serve the community if our program is successful.”


About DMD

Duchenne muscular dystrophy (DMD) is a disease that almost exclusively affects boys and whose incidence is 1 in 3,500. It is extremely rare that Duchenne muscular dystrophy (DMD) will affect girls. Those affected are usually diagnosed around the age of five, but symptoms may be visible from early childhood. It is a degenerative disease of the muscles caused by a genetic mutation. The Duchenne muscular dystrophy (DMD) – for which no treatment is currently available – directly affects skeletal muscles. Without treatment, the consequences of the disease are dire for those afflicted and their families.


About Sarepta Therapeutics

Sarepta Therapeutics is a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases. The Company is primarily focused on rapidly advancing the development of its potentially disease-modifying Duchenne muscular dystrophy (DMD) drug candidates. For more information, please visit www.sarepta.com.


More Links

La Force VLOG: Sarepta, grounded in the DMD community

Source: Sarepta Therapeutics, Inc.

Press release from Sarepta Therapeutics: Sarepta news release

Clinical trial: clinicaltrials.gov

*Muscular Dystrophy News Today: Microdystrophin Gene Therapy Shows Promising Interim Results in Phase 1/2 Trial

La Force BLOG: Gene therapy and exon skipping

La Force BLOG: Gene replacement therapy

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