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Elamipretide for Barth Syndrome Survives FDA Advisory Vote

Source link : https://www.newshealth.biz/health-news/elamipretide-for-barth-syndrome-survives-fda-advisory-vote/

FDA advisors indicated a possible path forward for elamipretide as a treatment for Barth syndrome despite the lack of definitive evidence. On Thursday, the Cardiovascular and Renal Drugs Advisory Committee voted 10-6 that elamipretide is effective for the ultra-rare cardioskeletal disease caused by TAFAZZIN gene mutations. Elamipretide is proposed as a first-in-class mitochondrial protective agent […]

Author : News Health

Publish date : 2024-10-11 13:21:54

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