Effect of rapid cessation of enzyme replacement therapy: a report of 5 more cases.
Department of Medical Genetics, The Children's Memorial Health Institute, Warsaw, Poland; Department of Molecular Biology, University of Gdańsk, Gdańsk, Poland. Electronic address: ajurecka@gmail.com.
Agnieszka
A
Jurecka
Department of Pediatrics and Adolescence Medicine, First Faculty of Medicine, Charles University, General University Hospital, Prague, Czech Republic.
Vera
V
Malinova
Department of Metabolic Diseases, The Children's Memorial Health Institute, Warsaw, Poland. Electronic address: a.tylki@czd.pl.
Anna
A
Tylki-Szymańska
D000293:Adolescent; D002648:Child; D018450:Disease Progression; D004334:Drug Administration Schedule; D056947:Enzyme Replacement Therapy; D005061:Europe, Eastern; D005260:Female; D006801:Humans; D007275:Injections, Intravenous; D008297:Male; D009083:Mucopolysaccharidoses / Q000473:pathology / Q000628:therapy*; D020051:N-Acetylgalactosamine-4-Sulfatase / Q000627:therapeutic use*; D011994:Recombinant Proteins / Q000627:therapeutic use; D028761:Withholding Treatment / Q000191:economics / Q000941:ethics*
D011994:Recombinant Proteins; D020051:N-Acetylgalactosamine-4-Sulfatase; C508864:galsulfase
Enzyme replacement therapy; Hunter syndrome; Maroteaux–Lamy syndrome; Mucopolysaccharidosis type II; Mucopolysacchridosis type VI