Browsing Tag
Niemann-Pick Disease
2 posts
IntraBio files first-ever FDA application for an Ataxia-Telangiectasia drug with AQNEURSA sNDA submission
IntraBio filed the first-ever FDA application for Ataxia-Telangiectasia via an AQNEURSA sNDA. Read what the Phase III data means for patients and the rare disease market.
Zevra Therapeutics’ MIPLYFFA receives FDA approval as first treatment for Niemann-Pick disease
Zevra Therapeutics Inc. has announced U.S. Food and Drug Administration (FDA) approval of MIPLYFFA (arimoclomol), a capsule-based treatment…