2024 Approvals
Miplyffa: A New Treatment for Niemann-Pick Disease Type C Approved on September 20, 2024
What is the name of the drug and what does it do? Miplyffa (pronounced MYE-pli-fah), generic name arimoclomol, is a medication used to treat neurological symptoms of Niemann-Pick disease type C (NPC). NPC is a rare genetic disorder that affects the body’s ability to process cholesterol and other fats,