Neuroradiological brain phenotype in mucopolysaccharidosis type II patients from 5 European countries
Igor Nestrašil(University of Minnesota), Martin Magner(Charles University), Vladimı́r Bzdúch(Comenius University Bratislava), Carol Nguyen(University of Minnesota), J Zeman(General University Hospital in Prague), Lenka Murgašová(Charles University), Katarína Juríčková(Comenius University Bratislava), Ivo Barić(University Hospital Centre Zagreb), Anna Tylki‐Szymańska(Children's Memorial Health Institute), Andrea Burgetová(Charles University), Zsuzsanna Almássy(Boston Children's Hospital), Danijela Petković Ramadža(University Hospital Centre Zagreb), Manuela Vaněčková
Cited by 0
Related Papers
Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy
|European Journal of Pediatrics|2007|535
Recommendations for initiation and cessation of enzyme replacement therapy in patients with Fabry disease: the European Fabry Working Group consensus document
|Orphanet Journal of Rare Diseases|2015|345
Elevated plasma glucosylsphingosine in Gaucher disease: relation to phenotype, storage cell markers, and therapeutic response
|Blood|2011|288
The phenotypic spectrum of organic acidurias and urea cycle disorders. Part 1: the initial presentation
|Journal of Inherited Metabolic Disease|2015|248
A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency
|New England Journal of Medicine|2015|244