Indication
Batten Disease
5 clinical trials
5 products
Clinical trial
Phase I/IIa Gene Transfer Clinical Trial for Juvenile Neuronal Ceroid Lipofuscinosis, Delivering the CLN3 Gene by Self-Complementary AAV9Status: Active (not recruiting), Estimated PCD: 2024-09-30
Product
AT-GTX-502Product
BMN190Clinical trial
A Phase 2, Open-Label, Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Intracerebroventricular BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 DiseaseStatus: Completed, Estimated PCD: 2022-04-20
Product
AT-GTX-501Clinical trial
Long-Term Follow-Up of AT-GTX-501 scAAV9 Gene Transfer in Subjects With CLN6 Batten DiseaseStatus: Active (not recruiting), Estimated PCD: 2024-10-01
Clinical trial
Longitudinal Study of Neurodegenerative DisordersStatus: Recruiting, Estimated PCD: 2030-01-01
Clinical trial
Augmentation of Umbilical Cord Blood Transplantation for Inherited Metabolic Diseases With Intrathecal Administration of Human Umbilical Cord Blood-Derived Oligodendrocyte-Like CellsStatus: Recruiting, Estimated PCD: 2024-10-01
Product
DUOC-01