Gene Therapy for Children With CLN3 Batten Disease

计划入组 7 人 · 首次发布于 2018 年 12 月 10 日

Natural History Study of Batten Disease

计划入组 10 人 · 首次发布于 2020 年 11 月 25 日

An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease

计划入组 6 人 · 首次发布于 2021 年 12 月 30 日

3rd Batten(Neurogene)

AT-GTX-502

liver disease program

Beyond Batten Disease Foundation

United States

Rare Disease Research, LLC

CASTLEMAN DISEASE COLLABORATIVE NETWORK

United States

New Retina Model Reveals How CLN3-Batten Disease Damages Vision and Points to a Treatment

Batten-1 Advances Toward Pivotal Phase 3 Trial as THX Pharma, Biocodex, and BBDF Unite on International Batten Disease Awareness Day

Azafaros to Present Phase 3 Nizubaglustat Data for Rare Lysosomal Storage Disorders at WORLDSymposium 2026