Biomarkers for prediction of skeletal disease progression in mucopolysaccharidosis type I-Reference-Cited by-同舟云学术

Biomarkers for prediction of skeletal disease progression in mucopolysaccharidosis type I

Published:2020-12-08 Issue:1 Volume:58 Page:89-99
ISSN:2192-8312
Container-title:JIMD Reports
language:en
Short-container-title:JIMD Reports

Author:

Lund Troy C.¹,Doherty Terence M.²,Eisengart Julie B.¹,Freese Rebecca L.³,Rudser Kyle D.⁴,Fung Ellen B.⁵,Miller Bradley S.¹,White Klane K.⁶,Orchard Paul J.¹,Whitley Chester B.¹,Polgreen Lynda E.²^ORCID

Affiliation:

1. Department of Pediatrics University of Minnesota Minneapolis Minnesota USA

2. Department of Pediatrics The Lundquist Institute at Harbor‐UCLA Medical Center Torrance California USA

3. Biostatistical Design and Analysis Center, Clinical and Translational Science Institute University of Minnesota Minneapolis Minnesota USA

4. School of Public Health, Division of Biostatistics University of Minnesota Minneapolis Minnesota USA

5. Department of Hematology University of California, San Francisco Benioff Children's Hospital Oakland California USA

6. Department of Orthopaedics and Sports Medicine Seattle Children's Hospital Seattle Washington USA

Funder

National Center for Advancing Translational Sciences

National Institute of Arthritis and Musculoskeletal and Skin Diseases

National Institute of Neurological Disorders and Stroke

Sanofi Genzyme

Publisher

Wiley

Subject

Biochemistry, Genetics and Molecular Biology (miscellaneous),Endocrinology, Diabetes and Metabolism,Internal Medicine

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/jmd2.12190

Reference55 articles.

1. The MPS I registry: Design, methodology, and early findings of a global disease registry for monitoring patients with Mucopolysaccharidosis Type I

2. Long-term outcome of Hurler syndrome following bone marrow transplantation

3. Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study

4. Long-term Outcomes of Adaptive Functions for Children with Mucopolysaccharidosis I (Hurler Syndrome) Treated with Hematopoietic Stem Cell Transplantation

5. Enzyme Replacement is Associated with Better Cognitive Outcomes after Transplant in Hurler Syndrome

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