"Promising Drug Slows Progression of Rare, Painful Genetic Disease"

1 min read
Source: National Institutes of Health (NIH) (.gov)
"Promising Drug Slows Progression of Rare, Painful Genetic Disease"
Photo: National Institutes of Health (NIH) (.gov)
TL;DR Summary

A drug used to treat certain bone diseases, etidronate, has shown promise in slowing the progression of ACDC disease, a rare genetic condition that causes excessive calcium buildup in the arteries. The first-in-human clinical trial, supported by the National Heart, Lung, and Blood Institute, found that the drug appeared safe and slowed the progression of new calcium deposits in the blood vessels of the legs, as well as improved symptoms such as pain and motion impairment. While it did not reverse existing calcium deposits or show clear improvement in blood flow, the study could lead to the first effective treatment for this rare disease and shed light on other diseases involving excessive calcium buildup in the arteries.

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