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Duchenne muscular dystrophy is the most common, severe, and life-threatening form of the degenerative neuromuscular disease affecting only boys, and the FDA just approved a drug that could change the lives of boys with the disease, NBC News reports.
Vamorolone, developed at Children's National Hospital in Washington, DC, is a steroidal-type, anti-inflammatory drug that has been shown in clinical trials to improve muscle strength and stature in boys with DMD without the side effects of steroids, such as weakened bones and stunted growth.
"Throughout my career, I have treated children with DMD, and I have seen over time how their shorter heights and brittle bones impact them physically and emotionallyin terms of their self-esteem and ability to participate in activities," the hospital's then-chief academic officer says in a press release.
"This drug should help these boys function more effectively and prevent certain long-term complications."
Vamorolone was developed using a different business model and drug development approach, including partnerships with the National Institutes of Health, the Department of Defense, the European Commission, and more than a dozen international nonprofit foundations.
In clinical trials, daily treatment with vamorolone improved muscle strength and stature with results comparable to prednisolone, but without
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