Lysosomal Storage Diseases
Azafaros Raises €132M in Series B to Fund Phase 3 Trials for Brain-Penetrant Drug
Azafaros; Series B funding; nizubaglustat; brain-penetrant drug; lysosomal storage disorders; rare neuro-metabolic diseases; phase 3 clinical trials; Jeito Capital; Forbion Growth; neurodegenerative diseases
Zevra Therapeutics Secures FDA Approval for Miplyffa, a Breakthrough Treatment for Niemann-Pick Disease Type C
Zevra Therapeutics, Miplyffa, FDA Approval, Niemann-Pick Disease Type C, Rare Lysosomal Storage Disorder, Arimoclomol