Consensus statement on enzyme replacement therapy for mucopolysaccharidosis IVA in Central and South-Eastern European countries
Martin Magner(Charles University), Christina Lampe(Justus-Liebig-Universität Gießen), Anna Tylki‐Szymańska(Children's Memorial Health Institute), Beata Kieć‐Wilk(Jagiellonian University), Dimitrios Zafeiriou(Aristotle University of Thessaloniki), Vasilica Plăiaşu(Institutul National pentru Sanatatea Mamei si Copilului "Alessandrescu-Rusescu"), Zoran Gucev, Zsuzsanna Almássy(Boston Children's Hospital), Ioannis Zaganas(University of Crete)
Cited by 6
Related Papers
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
<i>TBX6</i> Null Variants and a Common Hypomorphic Allele in Congenital Scoliosis
|New England Journal of Medicine|2015|317
Elevated plasma glucosylsphingosine in Gaucher disease: relation to phenotype, storage cell markers, and therapeutic response
|Blood|2011|288
Fourteen Monogenic Genes Account for 15% of Nephrolithiasis/Nephrocalcinosis
|Journal of the American Society of Nephrology|2014|243
Copy-Number Disorders Are a Common Cause of Congenital Kidney Malformations
|The American Journal of Human Genetics|2012|239