Orphan Drugs for Inherited Metabolic Diseases

计划入组 39 人 · 首次发布于 2023 年 4 月 19 日

Methadone Expansion in Detention Though the Hospital/Clinic Designation

计划入组 30 人 · 首次发布于 2026 年 7 月 16 日

Study of Genetic Polymorphisms of Drug Transporters and Orphan Nuclear Receptors on Treatment Effects of Irinotecan

计划入组 100 人 · 首次发布于 2007 年 7 月 25 日

Opaganib

C₂₃H₂₅ClN₂O

Ibudilast

C₁₄H₁₈N₂O

JUV-161

SWEDISH ORPHAN BIOVITRUM

France

Orphan Europe SARL

France

Swedish Orphan Biovitrum AG

Switzerland

uniQure's AMT-191 Receives FDA Orphan Drug Designation for Fabry Disease

EMA Grants Orphan Drug Designation to Devimistat for Refractory Burkitt's Lymphoma

Namodenoson Receives FDA Orphan Drug Designation for Pancreatic Cancer