Modern Approaches to the Management of Children with Mucopolysaccharidosis Type I-Reference-Cited by-同舟云学术

Modern Approaches to the Management of Children with Mucopolysaccharidosis Type I

Published:2022-10-04 Issue:4 Volume:19 Page:342-353
ISSN:2500-3089
Container-title:Pediatric pharmacology
language:
Short-container-title:Pediatr. farmakol.

Author:

Baidakova Galina V.¹,Baranov Alexander A.²,Vakhlova Irina V.³,Vashakmadze Nato D.⁴,Voskoboeva Elena Yu.¹,Zakharova Ekaterina Yu.¹,Kuzenkova Ludmila M.⁵,Kutsev Sergey I.¹,Larionova Valentina I.⁶,Lobzhanidze Tina V.⁷,Mikhailova Ludmila K.⁸,Mikhailova Svetlana V.⁹,Moiseev Sergey V.¹⁰,Namazova-Baranova Leyla S.¹¹,Nikitin Sergey S.¹²,Pechatnikova Natalya L.¹³,Polyakova Olga A.⁸,Semyachkina Alla N.¹⁴,Udalova Olga V.¹⁵

Affiliation:

1. Medical Genetic Research Center named after N.P. Bochkov

2. Pediatrics and Child Health Research Institute in Petrovsky National Research Centre of Surgery; Sechenov First Moscow State Medical University

3. Ural State Medical University

4. Pediatrics and Child Health Research Institute in Petrovsky National Research Centre of Surgery; Pirogov Russian National Research Medical University

5. Sechenov First Moscow State Medical University; National Medical Research Center for Children's Health

6. Institute of Experimental Education

7. City clinical hospital № 64

8. National Medical Research Center for Traumatology and Orthopedics named after N.N. Priorov

9. Russian Children’s Clinical Hospital

10. Sechenov First Moscow State Medical University

11. Pediatrics and Child Health Research Institute in Petrovsky National Research Centre of Surgery; Pirogov Russian National Research Medical University; Belgorod National Research University

12. Association of neuro-muscular disorders specialists

13. Morozov Children’s City Hospital

14. Veltischev Research and Clinical Institute for Pediatrics and Pediatric Surgery

15. Privolzhsky Research Medical University

Abstract

This article presents modern data on epidemiology, etiology, and clinical manifestations of mucopolysaccharidosis (MPS) type I in children. MPS develops due to deficiency of particular lysosomal enzyme which determines the disease type. The article considers in details disease's pathogenesis and classification. Evidence-based approaches to diagnosis (differential diagnosis included) are covered, moreover, special attention is paid to pathogenetic, symptomatic, and surgical treatment of MPS.

Publisher

Paediatrician Publishers LLC

Subject

Pulmonary and Respiratory Medicine,Pediatrics, Perinatology and Child Health

Reference45 articles.

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