The role of enzyme replacement therapy in severe Hunter syndrome—an expert panel consensus
Joseph Muenzer(University of North Carolina at Chapel Hill), Paul Harmatz(Royal Manchester Children's Hospital), Maurizio Scarpa(University of Udine), Simon Jones(Manchester Academic Health Science Centre), Gudrun Schulze Frenking(Johannes Gutenberg University Mainz), Olaf A. Bodamer(Universitätsklinik für Kinder und Jugendpsychiatrie), María Verónica Muñoz Rojas, Roberto Giugliani(Instituto de Biologia Molecular e Celular), Michael Beck(Universitätskinderklinik), Barbara K. Burton(Women & Children's Hospital of Buffalo), L. Clarke(University of British Columbia)
Cited by 112
Related Papers
Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease
|New England Journal of Medicine|2019|803
Phenylalanine hydroxylase deficiency: diagnosis and management guideline
|Genetics in Medicine|2014|718
Treatment of Fabry’s Disease with the Pharmacologic Chaperone Migalastat
|New England Journal of Medicine|2016|555
Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy
|European Journal of Pediatrics|2007|522
Enzyme replacement therapy for mucopolysaccharidosis VI: A phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study
|The Journal of Pediatrics|2006|364