Polycystic Ovary Syndrome in Type 1 Diabetes

计划入组 60 人 · 首次发布于 2026 年 3 月 24 日

Growth Curves in Cockayne Syndrome Type 1 and Type 2

计划入组 85 人 · 首次发布于 2021 年 10 月 25 日

Ultrasound Guided Diuretic Therapy in Type 1 Cardiorenal Syndrome

计划入组 80 人 · 首次发布于 2023 年 10 月 3 日

HSV-1 based gene therapy (Usher syndrome type 1)

Poliovirus type 1 antigen (formaldehyde inactivated)(Ipol, Kinrix, Pediarix, Pentacel, Quadracel, Vaxelis)

ALPORT SYNDROME FOUNDATION

United States

NHI-1

United States

International Rett Syndrome Foundation

United States

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

IDefine Partners with UT Southwestern to Develop First Gene Therapy for Kleefstra Syndrome

AAVantgarde Reports Positive Phase 1/2 Data for Gene Therapy AAVB-081 in Usher Syndrome Type 1B