Mucopolysaccharidosis I (MPS I) Registry

计划入组 1,500 人 · 首次发布于 2005 年 9 月 5 日

A Study of JR-171 in Patients With Mucopolysaccharidosis I

计划入组 18 人 · 首次发布于 2020 年 1 月 13 日

Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease

计划入组 3 人 · 首次发布于 2005 年 11 月 24 日

Mucopolysaccharidosis I therapeutic (bluebird bio)

IDUA gene therapy (AAV9, mucopolysaccharidosis I)

Mucopolysaccharidosis type I gene Therapy(Cellectis)

Universitetet i Oslo

Norway

I-SHOU UNIVERSITY

Taiwan

i-SENS, Inc.

South Korea

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