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4 studies found for:    CEROID LIPOFUSCINOSIS, NEURONAL, 1
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Rank Status Study
1 Completed A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease
Conditions: Jansky-Bielschowsky Disease;   Batten Disease;   Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2;   CLN2 Disease
Intervention: Biological: BMN 190
2 Completed Study of HuCNS-SC Cells in Patients With Infantile or Late Infantile Neuronal Ceroid Lipofuscinosis (NCL)
Condition: Neuronal Ceroid Lipofuscinosis
Interventions: Procedure: Surgery to implant human CNS stem cells (HuCNS-SC);   Drug: Medication to suppress the immune system
3 Completed
Has Results
Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis
Condition: Infantile Neronal Ceroid Lipofuscinosis
Intervention: Drug: Cystagon
4 Completed
Has Results
Stem Cell Transplant for Inborn Errors of Metabolism
Conditions: Adrenoleukodystrophy;   Metachromatic Leukodystrophy;   Globoid Cell Leukodystrophy;   Gaucher's Disease;   Fucosidosis;   Wolman Disease;   Niemann-Pick Disease;   Batten Disease;   GM1 Gangliosidosis;   Tay Sachs Disease;   Sandhoff Disease
Interventions: Procedure: Stem Cell Transplant;   Drug: Busulfan, Cyclophosphamide, Antithymocyte Globulin

Study has passed its completion date and status has not been verified in more than two years.