Zevra Therapeutics to Present New Long-Term Real-World Data on MIPLYFFA® in Niemann-Pick Disease Type C (NPC) at the 2026 SSIEM Annual Symposium
Zevra Therapeutics announced the presentation of long-term real-world data on its approved drug MIPLYFFA® (arimoclomol) for treating Niemann-Pick Disease Type C (NPC). The data underscore sustained disease stabilization across diverse patient populations up to 8 years, with favorable safety profile and no treatment-related discontinuations. These f…