Gene-Modified Stem Cell Therapy for Children With Autosomal Recessive Osteopetrosis (ARO)

计划入组 8 人 · 首次发布于 2026 年 6 月 24 日

Lymphocyte Phenotype of Autosomal Recessive Congenital Ichthyoses Mutated NIPAL4 (Nipal4-nEDD)

计划入组 10 人 · 首次发布于 2026 年 3 月 17 日

Validation of the RADIAL Algorithm for Diagnosis of Autosomal Recessive Cerebellar Ataxia

计划入组 400 人 · 首次发布于 2020 年 2 月 7 日

autosomal dominant polycystic kidney disease therapeutics

Autosomal dominant polycystic kidney disease(Maze Therapeutics)

autosomal dominant polycystic kidney disease therapy, Healx

Abeona Therapeutics

United States

Regulus Therapeutics

United States

NEW MEXICO CLINICAL RESEARCH AND OSTEOPOROSIS CENTER, INC.

United States

Scientists Discover Critical Window for In Vivo Gene Therapy in Newborns

Biallelic EHMT2 Variants Identified in Second-Ever Case of Autosomal Recessive Kleefstra-Like Neurodevelopmental Syndrome

āshibio Licenses Vantictumab for Rare Bone Disease ADO2 in Strategic Pipeline Expansion