FDA approves two treatments for Niemann-Pick Disease
This week, the US Food and Drug Administration (FDA) has approved both Zevra Therapeutics’ Miplyffa (arimoclomol) and IntraBio’s Aqneursa (levacetylleucine) for Neimann-Pick disease, type C (NPC). Miplyffa, in combination with the enzyme inhibitor miglustat, is approved to treat neurological symptoms associated with NPC in adults and… Read More