Ruxolitinib as First Line Treatment in Primary Haemophagocytic Lymphohistiocytosis (R-HLH)

计划入组 20 人 · 首次发布于 2023 年 3 月 9 日

Splenectomy as a Treatment for Patient With Relapsed Haemophagocytic Lymphohistiocytosis of Unknown Etiology

计划入组 30 人 · 首次发布于 2016 年 8 月 10 日

Study to Investigate Safety, Efficacy of an Anti-IFNγ mAb in Children With Primary Haemophagocytic Lymphohistiocytosis

计划入组 45 人 · 首次发布于 2013 年 3 月 26 日

familial hypercholesterolemia program

VRX-LDLR

LeXis gene therapy (familial hypercholesterolemia)

The Familial Hypercholesterolemia Foundation

United States

Madrigal Pharmaceuticals

United States

FUNDACION HIPERCOLESTEROLEMIA FAMILIAR

Spain

Genomic newborn screening catches rare diseases before symptoms start, with early trials flagging treatable conditions in up to 4% of infants

ELA026 Granted Orphan Drug Designation by FDA for Hemophagocytic Lymphohistiocytosis

FDA Approves First Treatment for Primary Hemophagocytic Lymphohistiocytosis, Marking 24-Year Breakthrough