Projects per year
- 1 - 50 out of 125 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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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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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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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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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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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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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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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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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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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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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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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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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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A Randomized, Controlled, Multicentre, Phase IIb Safety
SHIRE HUMAN GENETIC THERAPIES, INC.
12/15/14 → 10/31/16
Project: Research project
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A Randomized, Placebo-Controlled, Blind-Start, Single
ULTRAGENYX PHARMACEUTICAL INC.
11/13/14 → 6/1/16
Project: Research project
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Lysosomal Disease Network
Whitley, C. B., Cloyd, J. C., Coles, L. D., Connett, J. E., Kartha, R., Nestrasil, I., Rudser, K., Shapiro, E. G. & Ziegler, R. S.
NIH NAT INST OF NEURO DISORDERS & STROKE
9/30/14 → 8/1/19
Project: Research project