45 results on '"Neuronal Ceroid-Lipofuscinoses"'
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2. Functional Near-Infrared Spectroscopy (fNIRS) Combined With Diffuse Correlation Spectroscopy (DCS) in Neurocognitive Disease as Compared to Healthy Neurotypical Controls
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3. Caregiving Networks Across Disease Context and the Life Course
4. ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program (ScreenPlus)
5. UCB Transplant of Inherited Metabolic Diseases with Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells (DUOC-01)
6. Cerliponase Alfa Observational Study in the US
7. NYSCF Scientific Discovery Biobank
8. Gene Therapy Study for Children With CLN5 Batten Disease (CLN5-200)
9. A Natural History Study of Neuronal Ceroid Lipofuscinosis Type 5 (CLN5)
10. An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of CLN3 Disease
11. Baby Detect : Genomic Newborn Screening
12. Safety, Tolerability, and Efficacy of PLX-200 in Patients With CLN3
13. Longitudinal Study of Neurodegenerative Disorders
14. Longitudinal Assessment of Atypical Tripeptidyl Peptidase 1 Enzyme Deficiency Patients
15. A First-in-Human Study in Pediatric Patients With Ocular CLN2 Disease
16. Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2
17. Gene Therapy for Children With CLN3 Batten Disease
18. Clinical and Neuropsychological Investigations in Batten Disease
19. Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease
20. A Safety, Tolerability, and Efficacy Study of Intracerebroventricular BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease
21. Inherited Retinal Degenerative Disease Registry (MRTR)
22. Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease
23. Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer
24. Human Placental-Derived Stem Cell Transplantation (HPDSC)
25. Natural History of Neuronal Ceroid Lipofuscinosis, Batten's CLN6 Diseae (Batten'sCLN6)
26. An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease
27. Trehalose in Subjects With Neuronal Ceroid Lipofuscinoses (3AL-CLN36)
28. Natural History Study of Batten Disease
29. A Retrospective, Natural History Study in Children With CLN2
30. An Observational Study in Children With CLN2 Batten Disease
31. Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database
32. Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL)
33. AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis
34. Genotype-Phenotype Correlations of Late Infantile Neuronal Ceroid Lipofuscinosis
35. Using NPT to Evaluate Providing PPC as ELNEC-PPC WBT for Nurses (ELNEC-PPC)
36. Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis
37. Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis (JUMP)
38. 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
39. Stem Cell Transplant for Inborn Errors of Metabolism
40. Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis
41. Genotype - Phenotype Correlations of LINCL
42. Neuronal Ceroid Lipofuscinosis and Associated Sleep Abnormalities
43. Collection of Cerebrospinal Fluid in Healthy Children
44. Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis
45. Study of HuCNS-SC Cells in Patients With Infantile or Late Infantile Neuronal Ceroid Lipofuscinosis (NCL)
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