Posted by Michael Wonder on 20 Sep 2024
FDA approves first treatment for Niemann-Pick disease, type C
20 September 2024 - Today, the US FDA approved Miplyffa (arimoclomol), an oral medication for the treatment of Niemann-Pick disease, type C.
Miplyffa, in combination with the enzyme inhibitor miglustat, is approved to treat neurological symptoms associated with Niemann-Pick disease, type C in adults and children 2 years of age and older.
