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2023
A phase I/II study on intracerebroventricular tralesinidase alfa in patients with Sanfilippo syndrome type B
Muschol, N., Koehn, A., von Cossel, K., Okur, I., Ezgu, F., Harmatz, P., de Castro Lopez, M. J., Couce, M. L., Lin, S. P., Batzios, S., Cleary, M., Solano, M., Nestrasil, I., Kaufman, B., Shaywitz, A. J., Maricich, S. M., Kuca, B., Kovalchin, J. & Zanelli, E., Jan 17 2023, In: Journal of Clinical Investigation. 133, 2, e165076.Research output: Contribution to journal › Article › peer-review
Open Access -
Dilated Optic Nerve Sheath in Mucopolysaccharidosis I: Common and Not Necessarily High Intracranial Pressure
Huang, S., Lund, T., Orchard, P., Gupta, A. & Nascene, D., Jan 1 2023, In: American Journal of Neuroradiology. 44, 1, p. 91-94 4 p.Research output: Contribution to journal › Article › peer-review
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Neurologic Recovery in MPS i and MPS II Mice by AAV9-Mediated Gene Transfer to the CNS after the Development of Cognitive Dysfunction
Podetz-Pedersen, K. M., Laoharawee, K., Singh, S., Nguyen, T. T., Smith, M. C., Temme, A., Kozarsky, K., McIvor, R. S. & Belur, L. R., Jan 1 2023, In: Human gene therapy. 34, 1-2, p. 8-18 11 p.Research output: Contribution to journal › Article › peer-review
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2022
Contribution of the innate and adaptive immune systems to aortic dilation in murine mucopolysaccharidosis type I
Braunlin, E., Abrahante, J. E., Mc Elmurry, R. T., Evans, M. D., Smith, M., Seelig, D., O'Sullivan, G., Tolar, J., Whitley, C. B. & McIvor, R. S., Mar 2022, In: Molecular Genetics and Metabolism. 135, 3, p. 193-205 13 p.Research output: Contribution to journal › Article › peer-review
1 Scopus citations -
Early Neonatal Cardiac Phenotype in Hurler Syndrome: Case Report and Literature Review
Pillai, N. R., Ahmed, A., Vanyo, T. & Whitley, C. B., Aug 2022, In: Genes. 13, 8, 1293.Research output: Contribution to journal › Article › peer-review
Open Access -
Identification of a novel fusion Iduronidase with improved activity in the cardiovascular system
Kim, S., Przybilla, M. J., Whitley, C. B., Ou, L., Al-Kofahi, M. & Jarnes, J. R., Dec 2022, In: Molecular Genetics and Metabolism Reports. 33, 100917.Research output: Contribution to journal › Article › peer-review
Open Access2 Scopus citations -
Longitudinal Natural History of Pediatric Subjects Affected with Mucopolysaccharidosis IIIB
Okur, I., Ezgu, F., Giugliani, R., Muschol, N., Koehn, A., Amartino, H., Harmatz, P., de Castro Lopez, M. J., Couce, M. L., Lin, S. P., Batzios, S., Cleary, M., Solano, M., Peters, H., Lee, J., Nestrasil, I., Shaywitz, A. J., Maricich, S. M., Kuca, B., Kovalchin, J., & 1 others , Oct 2022, In: Journal of Pediatrics. 249, p. 50-58.e2Research output: Contribution to journal › Article › peer-review
Open Access1 Scopus citations -
Quantifying medical manifestations in Hurler syndrome with the infant physical symptom score: associations with long-term physical and adaptive outcomes
Ahmed, A., Rudser, K., King, K. E., Eisengart, J. B., Orchard, P. J., Shapiro, E. & Whitley, C. B., May 2022, In: Molecular Genetics and Metabolism. 136, 1, p. 22-27 6 p.Research output: Contribution to journal › Article › peer-review
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Quantitative brain MRI morphology in severe and attenuated forms of mucopolysaccharidosis type I
Kovac, V., Shapiro, E. G., Rudser, K. D., Mueller, B. A., Eisengart, J. B., Delaney, K. A., Ahmed, A., King, K. E., Yund, B. D., Cowan, M. J., Raiman, J., Mamak, E. G., Harmatz, P. R., Shankar, S. P., Ali, N., Cagle, S. R., Wozniak, J. R., Lim, K. O., Orchard, P. J., Whitley, C. B., & 1 others , Feb 2022, In: Molecular Genetics and Metabolism. 135, 2, p. 122-132 11 p.Research output: Contribution to journal › Article › peer-review
2 Scopus citations -
Tralesinidase alfa enzyme replacement therapy prevents disease manifestations in a canine model of mucopolysaccharidosis type IIIB
Ellinwood, N. M., Valentine, B. N., Hess, A. S., Jens, J. K., Snella, E. M., Jamil, M., Hostetter, S. J., Jeffery, N. D., Smith, J. D., Millman, S. T., Parsons, R. L., Butt, M. T., Chandra, S., Egeland, M. T., Assis, A. B., Nelvagal, H. R., Cooper, J. D., Nestrasil, I., Mueller, B. A., Labounek, R., & 17 others , Sep 1 2022, In: The Journal of pharmacology and experimental therapeutics. 382, 3, p. 277-286 10 p.Research output: Contribution to journal › Article › peer-review
Open Access1 Scopus citations -
2021
A multicenter open-label extension study of intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A
Wijburg, F. A., Whitley, C. B., Muenzer, J., Gasperini, S., del Toro, M., Muschol, N., Cleary, M., Sevin, C., Shapiro, E. & Alexanderian, D., Sep 1 2021, In: Molecular Genetics and Metabolism. 134, 1-2, p. 175-181 7 p.Research output: Contribution to journal › Article › peer-review
Open Access3 Scopus citations -
Biomarkers for prediction of skeletal disease progression in mucopolysaccharidosis type I
Lund, T. C., Doherty, T. M., Eisengart, J. B., Freese, R. L., Rudser, K. D., Fung, E. B., Miller, B. S., White, K. K., Orchard, P. J., Whitley, C. B. & Polgreen, L. E., Mar 1 2021, In: JIMD Reports. 58, 1, p. 89-99 11 p.Research output: Contribution to journal › Article › peer-review
Open Access7 Scopus citations -
Chitotriosidase as a biomarker for gangliosidoses
Kim, S., Whitley, C. B. & Jarnes, J. R., Dec 2021, In: Molecular Genetics and Metabolism Reports. 29, 100803.Research output: Contribution to journal › Article › peer-review
Open Access2 Scopus citations -
Comparative Effectiveness of Intracerebroventricular, Intrathecal, and Intranasal Routes of AAV9 Vector Administration for Genetic Therapy of Neurologic Disease in Murine Mucopolysaccharidosis Type I
Belur, L., Romero, M., Lee, J., Podetz-Pedersen, K. M., Nan, Z., Riedl, M. S., Vulchanova, L., Kitto, K. F., Fairbanks, C. A., Kozarsky, K. F., Orchard, P. J., Frey, W. H., Low, W. C. & McIvor, R. S., May 10 2021, In: Frontiers in Molecular Neuroscience. 14, 618360.Research output: Contribution to journal › Article › peer-review
Open Access14 Scopus citations -
Differences in MPS I and MPS II disease manifestations
Hampe, C. S., Yund, B. D., Orchard, P. J., Lund, T. C., Wesley, J. & Scott Mcivor, R., Aug 1 2021, In: International journal of molecular sciences. 22, 15, 7888.Research output: Contribution to journal › Review article › peer-review
Open Access7 Scopus citations -
Long-term safety and clinical outcomes of intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A
Wijburg, F. A., Heap, F., Rust, S., de Ruijter, J., Tump, E., Marchal, J. P., Nestrasil, I., Shapiro, E., Jones, S. A. & Alexanderian, D., Dec 2021, In: Molecular Genetics and Metabolism. 134, 4, p. 317-322 6 p.Research output: Contribution to journal › Article › peer-review
Open Access2 Scopus citations -
Mucopolysaccharidosis type I: Current treatments, limitations and prospects for improvement
Hampe, C. S., Wesley, J., Lund, T. C., Orchard, P. J., Polgreen, L. E., Eisengart, J. B., McLoon, L. K., Cureoglu, S., Schachern, P. & McIvor, R. S., Feb 2021, In: Biomolecules. 11, 2, p. 1-25 25 p., 189.Research output: Contribution to journal › Review article › peer-review
Open Access17 Scopus citations -
Outcome After Cord Blood Transplantation Using Busulfan Pharmacokinetics-Targeted Myeloablative Conditioning for Hurler Syndrome
Lum, S. H., Orchard, P. J., Lund, T. C., Miller, W. P., Boelens, J. J. & Wynn, R., 2021, In: Biology of Blood and Marrow Transplantation. 27, 1Research output: Contribution to journal › Article › peer-review
4 Scopus citations -
The natural history of neurocognition in MPS disorders: A review
Shapiro, E. G. & Eisengart, J. B., May 1 2021, In: Molecular Genetics and Metabolism. 133, 1, p. 8-34 27 p.Research output: Contribution to journal › Review article › peer-review
Open Access25 Scopus citations -
2020
Abnormally increased carotid intima media-thickness and elasticity in patients with Morquio A disease
Wang, R. Y., Rudser, K. D., Dengel, D. R., Evanoff, N., Steinberger, J., Movsesyan, N., Garrett, R., Christensen, K., Boylan, D., Braddock, S. R., Shinawi, M., Gan, Q. & Montanõ, A. M., Mar 17 2020, In: Orphanet Journal of Rare Diseases. 15, 1, 73.Research output: Contribution to journal › Article › peer-review
Open Access4 Scopus citations -
A Highly Efficacious PS Gene Editing System Corrects Metabolic and Neurological Complications of Mucopolysaccharidosis Type I
Ou, L., Przybilla, M. J., Ahlat, O., Kim, S., Overn, P., Jarnes, J., O'Sullivan, M. G. & Whitley, C. B., Jun 3 2020, In: Molecular Therapy. 28, 6, p. 1442-1454 13 p.Research output: Contribution to journal › Article › peer-review
Open Access32 Scopus citations -
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 2020, In: Pediatric Research. 87, 1, p. 104-111 8 p.Research output: Contribution to journal › Article › peer-review
Open Access7 Scopus citations -
Dysostosis Multiplex in Human Mucopolysaccharidosis Type 1 H and in Animal Models of the Disease
Hampe, C. S., Polgreen, L. E., Lund, T. C. & McIvor, R. S., Aug 1 2020, In: Pediatric endocrinology reviews : PER. 17, 4, p. 317-326 10 p.Research output: Contribution to journal › Article › peer-review
1 Scopus citations -
Intrathecal enzyme replacement for cognitive decline in mucopolysaccharidosis type I, a randomized, open-label, controlled pilot study
Chen, A. H., Harmatz, P., Nestrasil, I., Eisengart, J. B., King, K. E., Rudser, K., Kaizer, A. M., Svatkova, A., Wakumoto, A., Le, S. Q., Madden, J., Young, S., Zhang, H., Polgreen, L. E. & Dickson, P. I., Feb 2020, In: Molecular Genetics and Metabolism. 129, 2, p. 80-90 11 p.Research output: Contribution to journal › Article › peer-review
11 Scopus citations -
Intravenous delivery for treatment of mucopolysaccharidosis type I: A comparison of AAV serotypes 9 and rh10
Belur, L. R., Podetz-Pedersen, K. M., Tran, T. A., Mesick, J. A., Singh, N. M., Riedl, M., Vulchanova, L., Kozarsky, K. F. & McIvor, R. S., Sep 2020, In: Molecular Genetics and Metabolism Reports. 24, 100604.Research output: Contribution to journal › Article › peer-review
Open Access12 Scopus citations -
Mucopolysaccharidosis Type I: A Review of the Natural History and Molecular Pathology
Hampe, C. S., Eisengart, J. B., Lund, T. C., Orchard, P. J., Swietlicka, M., Wesley, J. & McIvor, R. S., Aug 5 2020, In: Cells. 9, 8Research output: Contribution to journal › Review article › peer-review
Open Access30 Scopus citations -
Myelin and Lipid Composition of the Corpus Callosum in Mucopolysaccharidosis Type I Mice
Le, S. Q., Nestrasil, I., Kan, S. H., Egeland, M., Cooper, J. D., Elashoff, D., Guo, R., Tolar, J., Yee, J. K. & Dickson, P. I., Nov 2020, In: Lipids. 55, 6, p. 627-637 11 p.Research output: Contribution to journal › Article › peer-review
1 Scopus citations -
Reduced-Toxicity (BuFlu) Conditioning Is Better Tolerated but Has a Higher Second Transplantation Rate Compared to Myeloablative Conditioning (BuCy) in Children with Inherited Metabolic Disorders
Gupta, A., Downey, M., Shanley, R., Jennissen, C., Miller, W. P., Lund, T. C., Orchard, P. J. & Smith, A. R., Mar 2020, In: Biology of Blood and Marrow Transplantation. 26, 3, p. 486-492 7 p.Research output: Contribution to journal › Article › peer-review
Open Access11 Scopus citations -
2019
A longitudinal study of neurocognition and behavior in patients with Hurler-Scheie syndrome heterozygous for the L238Q mutation
Ahmed, A., Ou, L., Rudser, K., Shapiro, E., Eisengart, J., King, K. E., Chen, A., Dickson, P. & Whitley, C. B., Sep 2019, In: Molecular Genetics and Metabolism Reports. 20, 100484.Research output: Contribution to journal › Article › peer-review
Open Access3 Scopus citations -
Attention and corpus callosum volumes in individuals with mucopolysaccharidosis type i
King, K. E., Rudser, K. D., Nestrasil, I., Kovac, V., Delaney, K. A., Wozniak, J. R., Mueller, B. A., Lim, K. O., Eisengart, J. B., Mamak, E. G., Raiman, J., Ali, N., Cagle, S., Harmatz, P., Whitley, C. B. & Shapiro, E. G., May 14 2019, In: Neurology. 92, 20, p. E2321-E2328Research output: Contribution to journal › Article › peer-review
7 Scopus citations -
Comprehensive behavioral and biochemical outcomes of novel murine models of GM1-gangliosidosis and Morquio syndrome type B
Przybilla, M. J., Ou, L., Tăbăran, A. F., Jiang, X., Sidhu, R., Kell, P. J., Ory, D. S., O'Sullivan, M. G. & Whitley, C. B., Feb 2019, In: Molecular Genetics and Metabolism. 126, 2, p. 139-150 12 p.Research output: Contribution to journal › Article › peer-review
Open Access15 Scopus citations -
Foundation of the Newborn Screening Translational Research Network and its tools for research
Lloyd-Puryear, M., Brower, A., Berry, S. A., Brosco, J. P., Bowdish, B. & Watson, M. S., Jun 1 2019, In: Genetics in Medicine. 21, 6, p. 1271-1279 9 p.Research output: Contribution to journal › Review article › peer-review
Open Access16 Scopus citations -
Genotype-phenotype correlation of gangliosidosis mutations using in silico tools and homology modeling
Ou, L., Kim, S., Whitley, C. B. & Jarnes-Utz, J. R., Sep 2019, In: Molecular Genetics and Metabolism Reports. 20, 100495.Research output: Contribution to journal › Article › peer-review
Open Access8 Scopus citations -
Genotype-phenotype relationships in mucopolysaccharidosis type I (MPS I): Insights from the International MPS I Registry
Clarke, L. A., Giugliani, R., Guffon, N., Jones, S. A., Keenan, H. A., Munoz-Rojas, M. V., Okuyama, T., Viskochil, D., Whitley, C. B., Wijburg, F. A. & Muenzer, J., Oct 1 2019, In: Clinical Genetics. 96, 4, p. 281-289 9 p.Research output: Contribution to journal › Article › peer-review
Open Access38 Scopus citations -
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 2019, In: Molecular Genetics and Metabolism. 126, 2, p. 117-120 4 p.Research output: Contribution to journal › Article › peer-review
2 Scopus citations -
Intrathecal enzyme replacement for Hurler syndrome: biomarker association with neurocognitive outcomes
Eisengart, J. B., Pierpont, E. I., Kaizer, A. M., Rudser, K. D., 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., Nov 1 2019, In: Genetics in Medicine. 21, 11, p. 2552-2560 9 p.Research output: Contribution to journal › Article › peer-review
Open Access17 Scopus citations -
Intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A: A phase IIb randomized trial
Wijburg, F. A., Whitley, C. B., Muenzer, J., Gasperini, S., del Toro, M., Muschol, N., Cleary, M., Sevin, C., Shapiro, E., Bhargava, P., Kerr, D. & Alexanderian, D., Feb 2019, In: Molecular Genetics and Metabolism. 126, 2, p. 121-130 10 p.Research output: Contribution to journal › Article › peer-review
Open Access28 Scopus citations -
In-vivo cortical thickness estimation from high-resolution T1w MRI scans in healthy and mucopolysaccharidosis affected dogs
Labounek, R., Mai, K., Mueller, B., Ellinwood, N. M., DIckson, P. & Nestrasil, I., Jul 2019, 2019 41st Annual International Conference of the IEEE Engineering in Medicine and Biology Society, EMBC 2019. Institute of Electrical and Electronics Engineers Inc., p. 2848-2851 4 p. 8856826. (Proceedings of the Annual International Conference of the IEEE Engineering in Medicine and Biology Society, EMBS).Research output: Chapter in Book/Report/Conference proceeding › Conference contribution
2 Scopus citations -
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., Miller, W., Lund, T., Armenian, S., Arora, M., Orchard, P. & Bhatia, S., Feb 2019, In: Biology of Blood and Marrow Transplantation. 25, 2, p. 328-334 7 p.Research output: Contribution to journal › Article › peer-review
Open Access7 Scopus citations -
Post-transplant laronidase augmentation for children with Hurler syndrome: biochemical outcomes
Lund, T. C., Miller, W. P., Liao, A. Y., Tolar, J., Shanley, R., Pasquali, M., Sando, N., Bigger, B. W., Polgreen, L. E. & Orchard, P. J., Dec 1 2019, In: Scientific reports. 9, 1, 14105.Research output: Contribution to journal › Article › peer-review
Open Access4 Scopus citations -
Recommendations for the management of MPS IVA: 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 , Jun 13 2019, In: Orphanet Journal of Rare Diseases. 14, 1, 137.Research output: Contribution to journal › Article › peer-review
Open Access42 Scopus citations -
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 , May 29 2019, In: Orphanet Journal of Rare Diseases. 14, 1, 118.Research output: Contribution to journal › Article › peer-review
Open Access24 Scopus citations -
Treatment of thoracolumbar kyphosis in patients with mucopolysaccharidosis type I: Results of an international consensus procedure
Kuiper, G. A., Langereis, E. J., Breyer, S., Carbone, M., Castelein, R. M., Eastwood, D. M., Garin, C., Guffon, N., Van Hasselt, P. M., Hensman, P., Jones, S. A., Kenis, V., Kruyt, M., Van Der Lee, J. H., Mackenzie, W. G., Orchard, P. J., Oxborrow, N., Parini, R., Robinson, A., Schubert Hjalmarsson, E., & 2 others , Jan 18 2019, In: Orphanet Journal of Rare Diseases. 14, 1, 17.Research output: Contribution to journal › Article › peer-review
Open Access9 Scopus citations -
ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome
Ou, L., DeKelver, R. C., Rohde, M., Tom, S., Radeke, R., St. Martin, S. J., Santiago, Y., Sproul, S., Przybilla, M. J., Koniar, B. L., Podetz-Pedersen, K. M., Laoharawee, K., Cooksley, R. D., Meyer, K. E., Holmes, M. C., McIvor, R. S., Wechsler, T. & Whitley, C. B., Jan 2 2019, In: Molecular Therapy. 27, 1, p. 178-187 10 p.Research output: Contribution to journal › Article › peer-review
Open Access53 Scopus citations -
2018
Beneath the floor: Re-analysis of neurodevelopmental outcomes in untreated Hurler syndrome
Shapiro, E. G., Whitley, C. B. & Eisengart, J. B., May 11 2018, In: Orphanet Journal of Rare Diseases. 13, 1, 76.Research output: Contribution to journal › Article › peer-review
Open Access15 Scopus citations -
Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing
Laoharawee, K., DeKelver, R. C., Podetz-Pedersen, K. M., Rohde, M., Sproul, S., Nguyen, H. O., Nguyen, T., St. Martin, S. J., Ou, L., Tom, S., Radeke, R., Meyer, K. E., Holmes, M. C., Whitley, C. B., Wechsler, T. & McIvor, R. S., Apr 4 2018, In: Molecular Therapy. 26, 4, p. 1127-1136 10 p.Research output: Contribution to journal › Article › peer-review
Open Access80 Scopus citations -
Erratum: Publisher Correction: Fast, sensitive method for trisaccharide biomarker detection in mucopolysaccharidosis type 1 (Scientific reports (2018) 8 1 (3681))
Makino, E., Klodnitsky, H., Leonard, J., Lillie, J., Lund, T. C., Marshall, J., Nietupski, J., Orchard, P. J., Miller, W. P., Phaneuf, C., Tietz, D., Varban, M. L., Donovan, M. & Belenki, A., Mar 19 2018, In: Scientific reports. 8, 1, p. 4994 1 p., 4994.Research output: Contribution to journal › Comment/debate › peer-review
Open Access -
Fast, sensitive method for trisaccharide biomarker detection in mucopolysaccharidosis type 1
Makino, E., Klodnitsky, H., Leonard, J., Lillie, J., Lund, T. C., Marshall, J., Nietupski, J., Orchard, P. J., Miller, W. P., Phaneuf, C., Tietz, D., Varban, M. L., Donovan, M. & Belenki, A., Dec 1 2018, In: Scientific reports. 8, 1, 3681.Research output: Contribution to journal › Article › peer-review
Open Access2 Scopus citations -
Long-term outcomes of systemic therapies for Hurler syndrome: an international multicenter comparison
Eisengart, J. B., Rudser, K. D., Xue, Y., Orchard, P., Miller, W., Lund, T., Van der Ploeg, A., Mercer, J., Jones, S., Mengel, K. E., Gökce, S., Guffon, N., Giugliani, R., de Souza, C. F. M., Shapiro, E. G. & Whitley, C. B., Nov 1 2018, In: Genetics in Medicine. 20, 11, p. 1423-1429 7 p.Research output: Contribution to journal › Article › peer-review
Open Access38 Scopus citations -
Metabolic Syndrome and Cardiovascular Risk Factors after Hematopoietic Cell Transplantation in Severe Mucopolysaccharidosis Type I (Hurler Syndrome)
Braunlin, E., Steinberger, J., DeFor, T., Orchard, P. & Kelly, A. S., Jun 2018, In: Biology of Blood and Marrow Transplantation. 24, 6, p. 1289-1293 5 p.Research output: Contribution to journal › Article › peer-review
Open Access5 Scopus citations