Source link : https://www.newshealth.biz/health-news/first-drug-approved-for-niemann-pick-disease-type-c/
The FDA on Friday approved arimoclomol (Miplyffa) as the first treatment specifically for the genetic disorder Niemann-Pick disease type C (NPC), an ultra-rare and progressive neurodegenerative disease. Arimoclomol oral capsules are indicated for the treatment of neurological manifestations of NPC in adults and children ages 2 years and older in combination with miglustat, an enzyme […]
Author : News Health
Publish date : 2024-09-20 17:31:44
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