Growth in individuals with attenuated mucopolysaccharidosis type <scp>I</scp> during untreated and treated periods: Data from the <scp>MPS I</scp> registry-Reference-Cited by-同舟云学术

Growth in individuals with attenuated mucopolysaccharidosis type I during untreated and treated periods: Data from the MPS I registry

Published:2022-07-23 Issue:10 Volume:188 Page:2941-2951
ISSN:1552-4825
Container-title:American Journal of Medical Genetics Part A
language:en
Short-container-title:American J of Med Genetics Pt A

Author:

Polgreen Lynda E.¹^ORCID,Bay Luisa²,Clarke Lorne A.³^ORCID,Guffon Nathalie⁴,Jones Simon A.⁵,Muenzer Joseph⁶,Flores Ana Lorena⁷,Wilson Kathryn⁷,Viskochil David⁸^ORCID

Affiliation:

1. The Lundquist Institute at Harbor‐UCLA Medical Center Torrance California USA

2. Hospital Nacional de Pediatría J. P. Garrahan Ciudad Autónoma de Buenos Aires Buenos Aires Argentina

3. Department of Medical Genetics and the British Columbia Children's Hospital Research Institute University of British Columbia Vancouver British Columbia Canada

4. Centre de Référence des Maladies Héréditaires du Métabolisme Hôpital Femme Mère Enfant Lyon France

5. St Mary's Hospital, Manchester University Foundation Trust University of Manchester Manchester UK

6. Department of Pediatrics University of North Carolina at Chapel Hill Chapel Hill North Carolina USA

7. Sanofi Cambridge Massachusetts USA

8. Department of Pediatrics, Division of Medical Genetics University of Utah School of Medicine Salt Lake City Utah USA

Funder

Sanofi

Publisher

Wiley

Subject

Genetics (clinical),Genetics

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/ajmg.a.62910

Reference38 articles.

1. Early treatment with laronidase improves clinical outcomes in patients with attenuated MPS I: a retrospective case series analysis of nine sibships

2. The natural history of MPS I: global perspectives from the MPS I Registry

3. Impact of Enzyme Replacement Therapy on Linear Growth in Korean Patients with Mucopolysaccharidosis Type II (Hunter Syndrome)

4. Pathogenesis of skeletal and connective tissue involvement in the mucopolysaccharidoses: glycosaminoglycan storage is merely the instigator

5. DepartmentHHS. (2002).2000 CDC growth charts for the United States: Methods and development: Data from the National Examination Surveys and the National Health and Nutrition Examination Surveys.Vital and Health Statistics Series 11 No. 246. Hyattsville MD: National Center for Health Statistics.

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