Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI

计划入组 39 人 · 首次发布于 2003 年 8 月 25 日

Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI

首次发布于 2002 年 11 月 6 日

Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI

首次发布于 2002 年 11 月 8 日

lentiviral vector-transduced stem cell gene therapy (Fondazione Telethon)

N-acetylgalactosamine-6-sulfatase gene therapy (lentiviral vector, Morquio syndrome)

Elosulfase alfa(Vimizim)

Rx

6'Naps

Belgium

October 6 University

Egypt

N&N Pharmaceuticals, Inc.

United States

NICE Provisionally Approves BioMarin's Vimizim for Ultra-Rare Morquio Disease with Real-World Data Requirements

Advancing Personalized Medicine: The Rise of N-of-1 Therapies for Rare Diseases

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