MPS II

HYOGO, Japan, June 8, 2026 JCR Pharmaceuticals Co., Ltd. announced new clinical findings from its J-Brain Cargo® blood-brain barrier...

SOUTH SAN FRANCISCO, Calif., March 25, 2026 In a landmark regulatory milestone, Denali Therapeutics announced that the U.S. Food...

EGENXBIO Inc. (Nasdaq: RGNX) announced positive 12-month pivotal data from its Phase I/II/III CAMPSIITE® trial of RGX-121 (clemidsogene lanparvovec),...