Duchenne Muscular Dystrophy (DMD) is an disease where the muscle experiences “dystrophy,” meaning muscle loss. It is a recessive genetic disease that rests on the X chromosome.1 Since biological males only have one X chromosome (XY), they are more susceptible to experiencing symptoms of an X-based recessive chromosomal disease. Biological females have two “X” chromosomes (XX), so there is less of a chance of a recessive X chromosome disease physically manifesting. This is why DMD is often considered more common in men than women. As a genetic disease DMD is passed from parents to their children.
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Symptoms of DMD include muscle weakness, fatigue, falls, breathing problems, delayed speech development, stunted growth, and more. There is currently no cure for DMD, so the main goals of therapy include symptom reduction and increasing quality of life. Some classes of medication involved in supportive care are corticosteroids, ACE inhibitors, and beta-blockers, coupled with potential physical therapy, exercise, and surgery.2
All the drugs mentioned above are not specific to DMD; rather, they treat various other ailments and have been “repurposed” for use in DMD. Therefore, there is a high necessity for specific and tailored treatments for individuals with DMD. A team at (SUNY) Binghamton University presented findings of a new drug, Vamorolone, at the Muscular Dystrophy Association’s Scientific and Clinical Conference in 2021. Those findings demonstrated the safety and efficacy of the agent in treating DMD and found that it is safer than prednisone, a corticosteroid used to treat DMD symptoms.3 These findings revealed that XY individuals with DMD who are treated with Vamorolone have better scores when conducting the NorthStar Ambulatory Assessment (NSAA), time to climb 4 stairs velocity (TTCLIMB) test, as well as better mental health outcomes, compared to prednisone.
ReveraGen BioPharma and Santhera Pharmaceuticals own Vamorolone. The FDA recently accepted their New Drug Application (NDA) for Vamorolone, and if approved, it could be on the market by the end of 2023. This is a milestone for various DMD patients across the world, as Vamorolone may become the first-line, standard of care drug for DMD.
References
1https://rarediseases.info.nih.gov/diseases/6291/duchenne-muscular-dystrophy
2https://my.clevelandclinic.org/health/diseases/23538-duchenne-muscular-dystrophy-dmd
3https://www.neurologylive.com/view/vamorolone-efficacious-dmd-superior-to-prednisone-safety