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Mucopolysaccharidosis I Medicine & Life Sciences
Cell Transplantation Medicine & Life Sciences
Hematopoietic Stem Cell Transplantation Medicine & Life Sciences
Adrenoleukodystrophy Medicine & Life Sciences
Osteopetrosis Medicine & Life Sciences
Mucopolysaccharidoses Medicine & Life Sciences
Transplants Medicine & Life Sciences
Transplantation Medicine & Life Sciences

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Projects 2007 2024

Hematopoietic Stem Cell Transplantation
Safety
Iduronidase
Beauty
Clinical Studies
Adrenoleukodystrophy
Hematopoietic Stem Cell Transplantation
Therapeutics

Research Output 1987 2019

1 Citation (Scopus)

A report on state-wide implementation of newborn screening for X-linked Adrenoleukodystrophy

Wiens, K., Berry, S. A., Choi, H., Gaviglio, A., Gupta, A. O., Hietala, A., Kenney-Jung, D. L., Lund, T. C., Miller, W., Pierpont, R., Raymond, G., Winslow, H., Zierhut, H. & Orchard, P. J., Jul 1 2019, In : American Journal of Medical Genetics, Part A. 179, 7, p. 1205-1213 9 p.

Research output: Contribution to journalArticle

Open Access
Adrenoleukodystrophy
Newborn Infant
Adrenal Insufficiency
Lysophosphatidylcholines
Pedigree
Profilins
Adrenoleukodystrophy
Blood-Brain Barrier
Antibodies
Immune System Diseases

Hematopoietic cell transplantation for severe MPS I in the first six months of life: The heart of the matter

Braunlin, E. A., Miettunen, K., Lund, T. C., Luquette, M. & Orchard, P. J., Feb 1 2019, In : Molecular Genetics and Metabolism. 126, 2, p. 117-120 4 p.

Research output: Contribution to journalArticle

Cell Transplantation
Screening
Neuroimaging
Intensive care units
Blood

Intrathecal enzyme replacement for Hurler syndrome: biomarker association with neurocognitive outcomes

Eisengart, J., Pierpont, R., Kaizer, A. M., Rudser, K., King, K. E., Pasquali, M., Polgreen, L. E., Dickson, P. I., Le, S. Q., Miller, W. P., Tolar, J., Orchard, P. J. & Lund, T. C., Jan 1 2019, In : Genetics in Medicine.

Research output: Contribution to journalArticle

Open Access
Mucopolysaccharidosis I
Enzyme Replacement Therapy
Biomarkers
Cerebrospinal Fluid
Enzymes
1 Citation (Scopus)

Late Mortality after Allogeneic Blood or Marrow Transplantation for Inborn Errors of Metabolism: A Report from the Blood or Marrow Transplant Survivor Study-2 (BMTSS-2)

Wadhwa, A., Chen, Y., Holmqvist, A., Wu, J., Ness, E., Parman, M., Kung, M., Hageman, L., Francisco, L., Braunlin, E. A., Miller, W., Lund, T. C., Armenian, S., Arora, M., Orchard, P. J. & Bhatia, S., Feb 1 2019, In : Biology of Blood and Marrow Transplantation. 25, 2, p. 328-334 7 p.

Research output: Contribution to journalArticle

Inborn Errors Metabolism
Survivors
Transplantation
Bone Marrow
Transplants