The innovative technology behind some of the most successful COVID-19 vaccines is showing signs of success in treating a rare but dangerous metabolic disease. Researchers report today in that regular infusions of messenger RNA (mRNA) coding for a missing enzyme may have slashed the rate of life-threatening medical emergencies in a handful of people with propionic acidemia, a condition in which toxic compounds build up in the body and damage organs.
The results from Moderna are the first published clinical data showing that mRNA, used in vaccines to deliver viral proteins that provoke an immune response, could potentially work as a drug to replace a protein people lack
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New mRNA therapy shows promise in treating 'ultrarare' inherited diseaseEmily is a health news writer based in London, United Kingdom. She holds a bachelor's degree in biology from Durham University and a master's degree in clinical and therapeutic neuroscience from Oxford University. She has worked in science communication, medical writing and as a local news reporter while undertaking journalism training.
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Givinostat Shows Positive Effects in Treating Duchenne Muscular DystrophyResults from the phase 3 EPIDYS trial show that givinostat, a drug for the treatment of Duchenne muscular dystrophy (DMD), has shown positive effects in reducing decline of muscle function and strength compared to placebo. The trial's primary endpoint, the four-stair climb assessment, worsened in both the treatment and placebo groups, but the decline was significantly smaller with givinostat. These findings provide strong support for the use of givinostat in the treatment of DMD. The drug is currently awaiting approval from the US Food and Drug Administration (FDA).
Source: Medscape - 🏆 386. / 55 Read more »