A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

计划入组 353 人 · 首次发布于 2008 年 11 月 10 日

Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA

计划入组 3 人 · 首次发布于 2012 年 11 月 27 日

Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)

计划入组 15 人 · 首次发布于 2012 年 1 月 24 日

GALNS gene therapy (AAV8, Mucopolysaccharidosis IVA)

IVA-360

Iva annua pollen

Inventiva

Fox Ira

Israel

INA RESPOND

Indonesia

FDA Grants Rare Pediatric Disease and Orphan Drug Designations to Six Gene Therapies

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

NICE Recommends Vanzacaftor-Tezacaftor-Deutivacaftor for Cystic Fibrosis Treatment in England