Psychological Concomitants of Morquio Syndrome (The MAP Study)

计划入组 20 人 · 首次发布于 2012 年 12 月 19 日

Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them?

计划入组 18 人 · 首次发布于 2017 年 5 月 11 日

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

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

GALNS gene therapy (AAV8, Morquio A syndrome)

Leigh syndrome therapeutic(Leigh syndrome)

ALPORT SYNDROME FOUNDATION

United States

International Rett Syndrome Foundation

United States

RETT SYNDROME RESEARCH TRUST

United States

BC Children's Hospital Launches Clinical Trials Super Hub with Community Support

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