Advancements in Rare Disease Therapies: Zevra Therapeutics and Clene Inc. Updates
Update: 2025-08-29
Description
Zevra Therapeutics presents new data on MIPLYFFA targeting Niemann-Pick disease type C at a congress, while Clene Inc. focuses on neurodegenerative diseases with lead candidate CNM-Au8® for ALS, gearing up for a Phase 3 trial and having a late-stage MS program. Both companies offer innovative approaches to treating rare diseases with significant investment potential.
Comments
In Channel