Immune Biomarkers Related to Bone Pathology in Patients With Type 1 Gaucher Disease

计划入组 40 人 · 首次发布于 2019 年 8 月 14 日

Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy

计划入组 40 人 · 首次发布于 2019 年 5 月 15 日

Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1)

计划入组 33 人 · 首次发布于 2015 年 10 月 22 日

Eliglustat(Cerdelga)

C₂₃H₃₆N₂O₄

Taliglucerase alfa(Elelyso)

Velaglucerase alfa(Vpriv)

Rx

NHI-1

United States

Xi'an No.1 Hospital

China

ALPHA-1 FOUNDATION, INC.

United States

Spur Therapeutics to Present Two-Year Clinical Data for FLT201 Gene Therapy in Gaucher Disease at WORLDSymposium

Precision Genomic Profiling Reveals Multiple Molecular Diagnoses in Gaucher Disease Patients with Atypical Presentations

Yuhan's YH35995 Receives FDA Orphan Drug Designation for Gaucher Disease Treatment