Study for the Treatment for CLN7 Disease

计划入组 4 人 · 首次发布于 2021 年 2 月 3 日

A Single Center Prospective Study in an Estimated 570 Patients Who Underwent Genetic Screening at UZ Brussel in the Context of a Primary Cardiac Arrhythmia. Patients Showing a Variant Class 3,4 or 5 in SCN4A or CLCN1 Will Undergo a Clinical and Electrophysiological Review After IC.

计划入组 570 人 · 首次发布于 2025 年 9 月 19 日

Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database

计划入组 500 人 · 首次发布于 2020 年 11 月 3 日

CLCN7-based siRNA therapy

CLN7(Neurogene)

CLN7 gene therapy (Neurogene)

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