Projects per year
Search results
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Active
A Multicenter Multinational Observational Study of Children withHypochondroplasia
1/10/24 → 1/31/34
Project: Research project
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A Multicenter, Multinational, Observational Study to Characterize Growth in Children with Idiopathic ShortStature
1/2/24 → 1/31/34
Project: Research project
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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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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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Region 4 Genetics Collaborative
MICHIGAN PUBLIC HEALTH INSTITUTE, US Dept. of Health & Human Services HRSA
6/1/17 → 5/31/24
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