Chemically modified recombinant human sulfamidase (SOBI003) in mucopolysaccharidosis IIIA patients: Results from an open, non-controlled, multicenter study-Reference-Cited by-同舟云学术

Chemically modified recombinant human sulfamidase (SOBI003) in mucopolysaccharidosis IIIA patients: Results from an open, non-controlled, multicenter study

Published:2022-08 Issue:4 Volume:136 Page:249-259
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Harmatz Paul,Muenzer Joseph,Ezgü Fatih,Dalén Per,Huledal Gunilla,Lindqvist Daniel,Gelius Stefan Svensson,Wikén Margareta,Önnestam Kristin,Bröijersén Anders

Funder

Swedish Orphan Biovitrum

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

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3. Mucopolysaccharidosis type IIIA: clinical spectrum and genotype-phenotype correlations;Valstar;Ann. Neurol.,2010

4. Mucopolysaccharidosis type III (Sanfilippo syndrome) and misdiagnosis of idiopathic developmental delay, attention deficit/hyperactivity disorder or autism spectrum disorder;Wijburg;Acta Paediatr.,2013

5. A prospective natural history study of mucopolysaccharidosis type IIIA;Shapiro;J. Pediatr.,2016

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1. Focal lesions following intracerebral gene therapy for mucopolysaccharidosis IIIA;Annals of Clinical and Translational Neurology;2023-05-11

2. Proteomics: Potential techniques for discovering the pathogenesis of connective tissue diseases-interstitial lung disease;Frontiers in Immunology;2023-03-29

3. A phase I/II study on intracerebroventricular tralesinidase alfa in patients with Sanfilippo syndrome type B;Journal of Clinical Investigation;2023-01-17