Projects per year
- 1 - 50 out of 155 results
Search results
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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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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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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 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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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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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
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Pilot Study of AGT-m195 in GLB1 Knockout Mouse Model of GM1-Gangliosidosis
Whitley, C. B. & Ou, L.
4/1/19 → 5/31/20
Project: Research project
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An Open-Label, Single-Arm, Multicenter Study of Intracerebral Administration of Adeno-Associated Viral Vectors Serotype rh10 Carrying the Human N-sulfoglucosamine sulfohydrolase(SGSH) cDNA for the Treatment ofMucopolysaccharidosis Type IIIA
Whitley, C. B., Belani, K. G. & Eisengart, J.
1/25/19 → 11/4/23
Project: Research project
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A Prospective Non-interventional Study in Subjects with
Whitley, C. B. & Karachunski, P. I.
6/8/18 → 6/29/19
Project: Research project
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Inborn Errors of Metabolism Collaborative Stakeholder Ne
MICHIGAN PUBLIC HEALTH INSTITUTE, PATIENT-CENTERED OUTCOMES RESEARCH INST.
1/1/18 → 11/30/18
Project: Research project
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A Phase I, Multicenter, Open-label, Single-dose
Whitley, C. B., Belani, K. G., Eisengart, J. & Miller, B. S.
6/16/17 → 5/1/19
Project: Research project
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A phase 3 randomized, multicenter, multinational, double
Whitley, C. B., Karachunski, P. I. & Miller, B. S.
6/14/17 → 6/29/20
Project: Research project
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A Randomized, Placebo-Controlled, Blind-Start, Single-Cr
Whitley, C. B. & Miller, B. S.
ULTRAGENYX PHARMACEUTICAL INC.
6/1/17 → 11/1/17
Project: Research project
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The Clinical and Economic Burden of Illness of MPS II (H
SHIRE HUMAN GENETIC THERAPIES, INC.
4/1/17 → 1/1/18
Project: Research project
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A Phase 2/3, multicenter, randomized, double-blinded, pl
Whitley, C. B. & Miller, B. S.
2/13/17 → 2/28/22
Project: Research project
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A Long-Term Open-Label Treatment and Extension Study of
Whitley, C. B. & Miller, B. S.
ULTRAGENYX PHARMACEUTICAL INC.
2/16/16 → 10/31/19
Project: Research project
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An Open Label Extension of Study HGT-SAN-093 Evaluating
SHIRE HUMAN GENETIC THERAPIES, INC.
1/11/16 → 6/1/18
Project: Research project
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Gene Therapy for Metabolic Disorders
NIH NAT'L INST OF CHILD HEALTH/HUMAN DEV
1/1/16 → 12/31/17
Project: Research project
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Megalencephaly and segmental brain overgrowth in humans
Millen, K. J. & Dobyns, W. B.
National Institute of Neurological Disorders and Stroke
9/30/15 → 7/31/21
Project: Research project
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A Randomized, Controlled, Multicentre, Phase IIb Safety
SHIRE HUMAN GENETIC THERAPIES, INC.
12/15/14 → 10/31/16
Project: Research project