Optical Imaging in X-linked Disorders.

计划入组 118 人 · 首次发布于 2025 年 3 月 11 日

Burosumab in Children and Adolescents With X-linked Hypophosphatemia

计划入组 57 人 · 首次发布于 2020 年 6 月 5 日

Education & Care in RARE: Efficacy of Targeted Psychoeducational Intervention Among Pediatric Rare Disease Patients

计划入组 100 人 · 首次发布于 2024 年 12 月 11 日

LUNAR-RARE2

trimethylangelicin (Rare Partners)

TUN-X,X

Molecule X LLC

Mexico

SFJ Pharmaceuticals X, LTD.

X-Pert-Med GmbH

Germany

Global Genes Partners with Citizen Health to Power RARE-X Rare Disease Data Platform

Researchers Identify Two Drug Candidates for Ultra-Rare X-Linked Myopathy Using Zebrafish Model

FDA Grants Priority Review for CUTX-101 in Treatment of Menkes Disease