1980 …2024
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  • 27 Similar Profiles
Mucopolysaccharidosis I Medicine & Life Sciences
Mucopolysaccharidoses Medicine & Life Sciences
Pediatrics Medicine & Life Sciences
Heart Transplantation Medicine & Life Sciences
Glycosaminoglycans Medicine & Life Sciences
Transplants Medicine & Life Sciences
Coronary Vessels Medicine & Life Sciences
Mucopolysaccharidosis VI Medicine & Life Sciences

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Projects 2009 2024

Mucopolysaccharidosis VI
Homologous Transplantation
Intravenous Administration
galsulfase
Dystrophin
Cardiomyopathies

Research Output 1980 2019

1 Citation (Scopus)

Clinical trial of laronidase in Hurler syndrome after hematopoietic cell transplantation

Polgreen, L. E., Lund, T. C., Braunlin, E., Tolar, J., Miller, B. S., Fung, E., Whitley, C. B., Eisengart, J. B., Northrop, E., Rudser, K., Miller, W. P. & Orchard, P. J., Jan 1 2019, (Accepted/In press) In : Pediatric Research.

Research output: Contribution to journalArticle

Open Access
Mucopolysaccharidosis I
Cell Transplantation
Clinical Trials
Anti-Idiotypic Antibodies
Pharmaceutical Preparations
Cell Transplantation
Screening
Neuroimaging
Intensive care units
Blood
2 Citations (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

Recommendations for the management of MPS IVA: Systematic evidence- A nd consensus-based guidance

Akyol, M. U., Alden, T. D., Amartino, H., Ashworth, J., Belani, K., Berger, K. I., Borgo, A., Braunlin, E., Eto, Y., Gold, J. I., Jester, A., Jones, S. A., Karsli, C., Mackenzie, W., Marinho, D. R., McFadyen, A., McGill, J., Mitchell, J. J., Muenzer, J., Okuyama, T. & 9 others, Orchard, P. J., Stevens, B., Thomas, S., Walker, R., Wynn, R., Giugliani, R., Harmatz, P., Hendriksz, C. & Scarpa, M., Jun 13 2019, In : Orphanet Journal of Rare Diseases. 14, 1, 137.

Research output: Contribution to journalArticle

Open Access
Mucopolysaccharidoses
Consensus
N-acetylglucosamine-6-sulfatase
Guidelines
Delivery of Health Care

Recommendations for the management of MPS VI: Systematic evidence- and consensus-based guidance

Akyol, M. U., Alden, T. D., Amartino, H., Ashworth, J., Belani, K., Berger, K. I., Borgo, A., Braunlin, E., Eto, Y., Gold, J. I., Jester, A., Jones, S. A., Karsli, C., Mackenzie, W., Marinho, D. R., McFadyen, A., McGill, J., Mitchell, J. J., Muenzer, J., Okuyama, T. & 9 others, Orchard, P. J., Stevens, B., Thomas, S., Walker, R., Wynn, R., Giugliani, R., Harmatz, P., Hendriksz, C. & Scarpa, M., May 29 2019, In : Orphanet Journal of Rare Diseases. 14, 1, 118.

Research output: Contribution to journalArticle

Open Access
Mucopolysaccharidosis VI
Consensus
Patient Advocacy
Delivery of Health Care
N-Acetylgalactosamine-4-Sulfatase