Sulfur Amino Acid supplementation may hold promise in Muscular dystrophy

Duchenne muscular dystrophy is the most common genetic disease affecting muscle. While gene therapy holds promise in clinical trials, it is not yet in common use. New treatments are needed while gene therapies are perfected.
Sulfur Amino Acid supplementation may hold promise in Muscular dystrophy
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Recently we discovered a new drug that improves health in a worm model of Duchenne muscular dystrophy. This drug appears to improve health by increasing the amount of Sulphur in the muscles. More specifically, it increases the amount of Sulphur in the mitochondria, which are the power plant of the cell. We also found that mouse models of Duchenne muscular dystrophy have reduced amounts of Sulphur. This suggested that the new drug could work in animals and possibly patients. However, going from an initial discovery of a new drug to a drug that works well in patients is a long process. It often takes twenty years. Knowing that the drug gives Sulphur to the muscles to improve health, we asked if we could use a natural form of Sulphur to improve health. We chose the naturally occurring Sulphur Amino Acids, which are already present in our bodies. Feeding these Sulphur Amino Acids to the Duchenne muscular dystrophy worms did indeed increase the amount of Sulphur in the muscles and improve health. Feeding two of these Amino Acids to mouse models of Duchenne muscular dystrophy has also been shown to improve health. Thus, results suggest that feeding Sulphur Amino Acids to Duchenne muscular dystrophy patients may have benefit. However, more studies are needed to confirm this. Specifically, which patients can benefit and how much supplement they need are important unanswered questions. We think that Sulphur levels decrease with increased severity of the disease and therefore such supplementation approaches will need to be tailored to each patient. These are all questions that can now be addressed in patients rather than in the worm model.

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