Therapy development for the mucopolysaccharidoses: Updated consensus recommendations for neuropsychological endpoints-Reference-Cited by-同舟云学术

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Therapy development for the mucopolysaccharidoses: Updated consensus recommendations for neuropsychological endpoints

Published:2020-09 Issue:1-2 Volume:131 Page:181-196
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

van der Lee Johanna H.,Morton Jonathan,Adams Heather R.,Clarke Lorne,Eisengart Julie B.,Escolar Maria L.,Giugliani Roberto,Harmatz Paul,Hogan Melissa,Kearney Shauna,Muenzer Joseph,Muschol Nicole,Rust Stewart,Saville Benjamin R.,Semrud-Clikeman Margaret,Wang Raymond,Shapiro Elsa

Funder

National MPS Society

Abeona Therapeutics

Allievex

Amicus Therapeutics

BioMarin

bluebird bio

Denali Therapeutics

JCR Pharmaceutical

Orchard Therapeutics

ReGenXBio

Sanofi Genzyme

Seelos Therapeutics

Takeda

Ultragenyx

Aeglea Biotherapeutics

PTC Therapeutics

Shire

Publisher

Elsevier BV

Subject

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

Reference162 articles.

1. The mucopolysaccharidoses;Neufeld,2001

2. Why are behaviors of children suffering from various neuronopathic types of mucopolysaccharidoses different?;Wegrzyn;Med Hypotheses,2010

3. Developmental and behavioral aspects of mucopolysaccharidoses with brain manifestations - neurological signs and symptoms;Shapiro;Mol Genet Metab,2017

4. Behaviour in mucopolysaccharide disorders;Bax;Arch Dis Child,1995

5. Mucopolysaccharidosis III (Sanfilippo syndrome) - disease presentation and experimental therapies;Gilkes;Pediatr Endocrinol Rev,2014

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1. Genotype–phenotype findings in patients with mucopolysaccharidosis II from the Hunter Outcome Survey;Molecular Genetics and Metabolism;2024-09

2. Anakinra in Sanfilippo syndrome: a phase 1/2 trial;Nature Medicine;2024-06-21

3. Clinical investigator perspectives on patient outcomes in children with neuronopathic mucopolysaccharidosis II during intrathecal idursulfase-IT treatment;Orphanet Journal of Rare Diseases;2024-04-12

4. Caregiver experiences and observations of intrathecal idursulfase-IT treatment in a phase 2/3 trial in pediatric patients with neuronopathic mucopolysaccharidosis II;Orphanet Journal of Rare Diseases;2024-03-10

5. Ability change across multiple domains in mucopolysaccharidosis (Sanfilippo syndrome) type IIIA;Molecular Genetics and Metabolism;2024-02

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