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Expertise related to UN Sustainable Development Goals
In 2015, UN member states agreed to 17 global Sustainable Development Goals (SDGs) to end poverty, protect the planet and ensure prosperity for all. This person’s work contributes towards the following SDG(s):
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The Fingerprint is created by mining the titles and abstracts of the person's research outputs and projects/funding awards to create an index of weighted terms from discipline-specific thesauri.
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Collaborations and top research areas from the last five years
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An Open Label, Dose Range, Proof-of-Concept Study to Assess the Safety and Efficacy of HM15421/GC1134A in Patients with Fabry Disease
Whitley, C. B. (PI) & Miller, B. S. (CoI)
8/18/25 → 8/17/35
Project: Research project
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An Open-Label, Multiple-Center Phase I/II Dose Escalation Study for theSafety and Efficacy of NGGT002 in Adults with Classic Phenylketonuria
Whitley, C. B. (PI)
6/2/25 → 6/1/35
Project: Research project
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A Phase 1, Open-label, Ascending Dose Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of Recombinant Human Heparan N-Sulfatase (rhHNS, GC1130A) Via Intracerebroventricular Access Device in Patients with
Whitley, C. B. (PI)
2/1/25 → 1/31/35
Project: Research project
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A Phase 1/2, Single Dose, Dose Escalation Study of Intravenous AAV5-GLA (AMT-191) in Adult Males with Classic Fabry Disease
Whitley, C. B. (PI)
1/10/25 → 3/31/35
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. (PI), Diethelm-Okita, B. M. (CoI), Jarnes, J. R. (CoI) & Miller, B. S. (CoI)
12/4/23 → 11/30/30
Project: Research project
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Addressing the Multisystemic Impacts of Nephropathic Cystinosis in an Adult
Jarnes, J. R., Palmer, R. S. & Whitley, C. B., Mar 2025, In: Kidney International Reports. 10, 3, p. S789-S793Research output: Contribution to journal › Article › peer-review
Open Access -
Bone marrow transplantation reverses metabolic alterations in multiple sulfatase deficiency: a case series
Pillai, N. R., Liu, N., Li, X., Li, X., Ahrens-Nicklas, R., Adang, L., Eisengart, J. B., Bronken, G., Gupta, A., Lund, T. C., Whitley, C. B., Elsea, S. H. & Orchard, P. J., Dec 2025, In: Communications Medicine. 5, 1, 12.Research output: Contribution to journal › Article › peer-review
Open Access1 Scopus citations -
Detecting the Difficult: An Intronic NPC1 Variant Hiding in Plain Sight
Brown, C. G., Bower, M., Schomaker, M., Goldstein, J., Jarnes, J., Whitley, C. B. & Pillai, N. R., Jul 2025, In: American Journal of Medical Genetics, Part A. 197, 7, e64012.Research output: Contribution to journal › Article › peer-review
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Diffusion tensor imaging with free-water correction reveals distinctions between severe and attenuated subtypes in Mucopolysaccharidosis type I
Svatkova, A., Pasternak, O., Eisengart, J. B., Rudser, K. D., Bednařík, P., Mueller, B. A., Delaney, K. A., Shapiro, E. G., Whitley, C. B. & Nestrašil, I., Jan 2025, In: Journal of Inherited Metabolic Disease. 48, 1, e12830.Research output: Contribution to journal › Article › peer-review
Open Access -
Dose-intensive therapy (DIT) for infantile Pompe disease: A pilot study
Jarnes, J. R., Pillai, N. R., Ahmed, A., Shrestha, S., Stark, M. & Whitley, C. B., Mar 2025, In: Molecular Genetics and Metabolism Reports. 42, 101179.Research output: Contribution to journal › Article › peer-review
Open Access