Business Wire

GC Pharma Announces EMA Grants Orphan Drug Designation to Hunterase ICV, The World’s First Enzyme Replacement Therapy for Mucopolysaccharidosis Type II Administered by ICV ...

YONGIN, South Korea--(BUSINESS WIRE)--GC Pharma (006280.KS) today announced that the European Medicines Agency (EMA) granted orphan drug designation (ODD) to Hunterase ICV (intracerebroventricular, ...
GEN

Triplet’s New Huntington Disease Drug May Also Treat Other Repeat Expansion Disorders

In a virtual presentation at the CHDI Foundation’s 16 th Annual Huntington’s Disease (HD) Therapeutics Conference, Triplet Therapeutics presented preclinical data on the therapeutic target and route ...