Natural History Studies of Mucopolysaccharidosis III

计划入组 25 人 · 首次发布于 2014 年 1 月 16 日

A Study of JR-141 in Patients With Mucopolysaccharidosis II

计划入组 28 人 · 首次发布于 2018 年 6 月 26 日

Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)

计划入组 96 人 · 首次发布于 2003 年 10 月 1 日

SGSH gene therapy (AAV9, mucopolysaccharidosis IIIA)

AAV-TT coHGSNAT gene therapy (intracerebral, mucopolysaccharidosis IIIC)

triple capsid mutant based NAGLU gene therapy (AAV8, Mucopolysaccharidosis type IIIB)

Celgene International II SARL

Switzerland

Meiragtx UK II Ltd.

Belgium

Generics Bidco II, LLC

United States

JCR Pharmaceuticals' JR-441 Receives Clearance for Phase I MPS IIIA Trial in Japan

Denali's Hunter Syndrome Candidate Receives FDA Orphan Drug and Rare Pediatric Disease Designations

GC Biopharma's Hunterase ICV Approved in Russia for Severe Hunter Syndrome