Projects per year
- 1 - 50 out of 180 results
Search results
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Active
Long-Term Follow-up of Fabry Disease Subjects who have Undergone IV Infusion with ST-920, an rAAV2/6 Human Alpha Galactosidase A Gene Therapy
Whitley, C. B., Diethelm-Okita, B. M., Jarnes, J. R. & Miller, B. S.
12/4/23 → 11/30/30
Project: Research project
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Collection of Gene Mutations for Laboratory Quality Assurance: Newborn Screening Accuracy Project
SEQUOIA FOUNDATION, CENTERS FOR DISEASE CONTROL & PREVENTION
5/15/23 → 5/14/28
Project: Research project
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A Phase III study of JR-141 in Mucopolysaccharidosis type II (Hunter Syndrome) patients
Whitley, C. B. & Miller, B. S.
11/15/22 → 11/16/25
Project: Research project
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Transcriptomic Approaches to Diagnosis and Management of Inborn Errors of Immunity
Thielen, B. K., Albert, F. W. & Billington, C.
7/1/22 → 6/30/24
Project: Research project
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Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program (LC-FAOD DMP)
ULTRAGENYX PHARMACEUTICAL INC.
8/3/21 → 8/31/26
Project: Research project
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ACC: Callosal Agenesis as a Window into Common Neurodevelopmental Disorders
UNIVERSITY OF CALIFORNIA, SAN FRANCISCO, NIH NAT INST OF NEURO DISORDERS & STROKE
5/1/21 → 4/30/24
Project: Research project
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Lysosomal Disease Network
Whitley, C. B., Ahmed, A., Braunlin, E. A., Cortez, D., Eisengart, J., King, K. E., Nestrasil, I. & Orchard, P. J.
NIH NAT INST OF NEURO DISORDERS & STROKE
9/15/19 → 7/31/25
Project: Research project
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Hunter Syndrome Outcome Survey (HOS)
SHIRE HUMAN GENETIC THERAPIES, INC.
1/15/17 → 1/14/27
Project: Research project
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Mosaic: post-zygotic mutations in vascular malformations
Bennett, J. J. T., Dobyns, W. B. & Perkins, J. A.
National Heart, Lung, and Blood Institute
2/8/16 → 6/30/24
Project: Research project
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Lysosomal Disease Network
Whitley, C. B., Ballon, D. J., Bjoraker, K. J., Cathey, S. S., Chen, A. H., Cloyd, J. C., Crystal, R. G., Grabowski, G. A., Kishnani, P. S., Kishnani, P. S., Langan, T. J., Levy, P., Mauer, M. S., Mink, J. W., Najafian, B., Patterson, M. C., Polgreen, L. E., Schiffmann, R. & Shapiro, E. G.
National Institute of Neurological Disorders and Stroke
9/30/09 → 7/31/24
Project: Research project
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Rare Diseases Clinical Research Consortia (RDCRC) for the Rare Diseases Clinical Research Network (RDCRN) (U54 Clinical Trial Optional)
Gropman, A., Batshaw, M. L., Berry, S. A., Burrage, L. C., Le Mons, C., Lee, B., Nagamani, S., Sreenath-Nagamani, S. & Tuchman, M.
9/25/09 → 7/31/24
Project: Research project
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ACC: Callosal Agenesis as a Window into Common Neurodevelopmental Disorders
Dobyns, W. B. & Sherr, E. H.
National Institute of Neurological Disorders and Stroke
3/1/08 → 4/30/24
Project: Research project
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Not started
Advanced Therapies Rare Genetic Disease Fellowship
Jarnes, J. R. & Whitley, C. B.
7/1/24 → 6/30/25
Project: Research project
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Finished
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An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adult Males with Fabry Disease
3/30/22 → 3/1/23
Project: Research project
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Urea Cycle Disorders Consortium - Project(s) 1 (and 3, if applicable)
8/1/21 → 7/31/22
Project: Research project
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Protocol Development for Post-Market Surveillance of Carbaglu in Propionic Acidemia And Methylmalonic Acidemia
5/15/21 → 7/31/21
Project: Research project
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Safety and efficacy of Nov340 delivery of SaRNA in treating MPS I disease
Whitley, C. B., Lund, T. C., Orchard, P. J. & Ou, L.
5/1/21 → 4/29/23
Project: Research project
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The Genetic Basis of Dandy-Walker and Other Mid-Hindbrain Malformations
NIH NAT INST OF NEURO DISORDERS & STROKE
12/20/20 → 5/31/22
Project: Research project
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Brain connectome as a novel biomarker of Krabbe disease
Whitley, C. B. & Ou, L.
10/15/20 → 10/14/22
Project: Research project
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Inborn Errors of Metabolism Research Fund
CHILDREN'S RESEARCH INSTITUTE, KETTERING FDN
8/1/20 → 7/31/21
Project: Research project
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A Multi-Center, Low Interventional Study with a Retrospective Component in Participants with Late Onset Pompe Disease
12/17/19 → 1/31/21
Project: Research project
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Clinical-Res-Project1
Berry, S. A. & Berry, S. S. A.
National Institute of Child Health and Human Development
9/17/19 → 7/31/23
Project: Research project
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Lysosomal Disease Network Pilot Core
Whitley, C. B. & Whitley, C. C. B.
National Institute of Neurological Disorders and Stroke
8/1/19 → 7/31/22
Project: Research project
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Lysosomal Disease Network Administrative Core
Whitley, C. B. & Whitley, C. C. B.
National Institute of Neurological Disorders and Stroke
8/1/19 → 7/31/22
Project: Research project
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Career Enhancement Core
Whitley, C. B. & Whitley, C. C. B.
National Institute of Neurological Disorders and Stroke
8/1/19 → 7/31/22
Project: Research project
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MPS Disease Longitudinal Study of Treatment and Outcomes Across the Lifespan
Whitley, C. B. & Whitley, C. C. B.
National Institute of Neurological Disorders and Stroke
8/1/19 → 7/31/22
Project: Research project