Classic infantile Pompe patients approaching adulthood: a cohort study on consequences for the brain-Reference-Cited by-同舟云学术

Classic infantile Pompe patients approaching adulthood: a cohort study on consequences for the brain

Published:2018-03-24 Issue:6 Volume:60 Page:579-586
ISSN:0012-1622
Container-title:Developmental Medicine & Child Neurology
language:en
Short-container-title:Dev Med Child Neurol

Author:

Ebbink Berendine J¹,Poelman Esther¹,Aarsen Femke K¹,Plug Iris¹,Régal Luc²,Muentjes Carsten³,Beek Nadine A M E⁴,Lequin Maarten H⁵,Ploeg Ans T¹,Hout Johanna M P¹^ORCID

Affiliation:

1. Department of Pediatrics Center for Lysosomal and Metabolic Diseases Erasmus MC University Medical Center Rotterdam the Netherlands

2. Department of Pediatrics, Pediatric Neurology and Metabolic Disorders UZ Brussel Brussels Belgium

3. Department of Pediatrics III University Children′s Hospital Essen Germany

4. Department of Neurology Center for Lysosomal and Metabolic Diseases Erasmus MC University Medical Center Rotterdam the Netherlands

5. Division of Pediatric Radiology Department of Pediatrics Utrecht University Utrecht the Netherlands

Funder

Ministerie van Economische Zaken

Prinses Beatrix Spierfonds

Conselho Nacional de Desenvolvimento Científico e Tecnológico

Publisher

Wiley

Subject

Neurology (clinical),Developmental Neuroscience,Pediatrics, Perinatology and Child Health

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1111/dmcn.13740

Reference26 articles.

1. The Natural Course of Infantile Pompe’s Disease: 20 Original Cases Compared With 133 Cases From the Literature

2. Recombinant human α-glucosidase from rabbit milk in Pompe patients

3. Response of 33 UK patients with infantile-onset Pompe disease to enzyme replacement therapy

4. Outcome of Patients with Classical Infantile Pompe Disease Receiving Enzyme Replacement Therapy in Germany

5. Recombinant human acid -glucosidase: Major clinical benefits in infantile-onset Pompe disease

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