Zevra Therapeutics Presents Positive New Data Supporting Foundational Role of MIPLYFFA® (arimoclomol) for the Treatment of Niemann-Pick Disease Type C at the International Congress of Inborn Errors of Metabolism (ICIEM)
Zevra Therapeutics(US:ZVRA) Globenewswire·2025-09-04 19:30New data from pre-specified analysis shows patients on concomitant miglustat who switched from placebo to MIPLYFFA experienced a decline in annual disease progression Nomination for Best Poster Award received for poster highlighting MIPLYFFA’s differentiated mechanism of action targets underlying pathology of NPC CELEBRATION, Fla., Sept. 04, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living ...