Article

Breakthrough in Rare Diseases: FDA Approves Givinostat for Duchenne Muscular Dystrophy

2024-11-19

Abstract excerpt

A major development in the treatment of a rare genetic disease, duchenne muscular dystrophy, has recently been approved by the FDA. Givinostat, a histone-acetylase inhibitor, signifies a new ray of hope for patients with DMD, a progressive and enervating disease. This treatment, given as an oral suspension, was approved for use in patients 6 years and older after promising results were obtained from the phase 3 EP...

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Literature Corpus work: 340cb77c-5391-598b-95ec-33fd0423e3dc
DOI: 10.22541/au.173202872.20286590/v1

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