Intrathecal/Intracerebroventricular enzyme replacement therapy for the mucopolysaccharidoses: efficacy, safety, and prospects-Reference-Cited by-同舟云学术

Intrathecal/Intracerebroventricular enzyme replacement therapy for the mucopolysaccharidoses: efficacy, safety, and prospects

Published:2018-06-22 Issue:7 Volume:6 Page:403-411
ISSN:2167-8707
Container-title:Expert Opinion on Orphan Drugs
language:en
Short-container-title:Expert Opinion on Orphan Drugs

Author:

Giugliani Roberto¹²³⁴^ORCID,Dalla Corte Amauri⁴^ORCID,Poswar Fabiano¹^ORCID,Vanzella Cláudia¹^ORCID,Horovitz Dafne⁵^ORCID,Riegel Mariluce¹³^ORCID,Baldo Guilherme³⁶^ORCID,Vairo Filippo⁷^ORCID

Affiliation:

1. Medical Genetics Service, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil

2. Department of Genetics, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil

3. Postgraduate Program in Genetics and Molecular Biology, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil

4. Post-Graduate Program in Medical Sciences, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil

5. Department of Medical Genetics, National Institute for Women, Children and Adolescent Health Fernandes Figueira/Fiocruz, Rio de Janeiro, Brazil

6. Postgraduate Program in Physiology, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil

7. Center of Individualized Medicine, Mayo Clinic, Rochester, MN, USA

Funder

no funded

Publisher

Informa UK Limited

Subject

Pharmacology (medical),Health Policy,Pharmacology, Toxicology and Pharmaceutics (miscellaneous)

Link

https://www.tandfonline.com/doi/pdf/10.1080/21678707.2018.1487838

Reference55 articles.

1. New GLB1 mutation in siblings with Morquio type B disease presenting with mental regression

2. A complete deficiency of Hyaluronoglucosaminidase 1 (HYAL1) presenting as familial juvenile idiopathic arthritis

3. Mutations in HYAL1, a member of a tandemly distributed multigene family encoding disparate hyaluronidase activities, cause a newly described lysosomal disorder, mucopolysaccharidosis IX

4. Pre-transplant risk factors affecting outcome in Hurler syndrome

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