Effect of rapid cessation of enzyme replacement therapy: A report of 5 more cases

Molecular Genetics and Metabolism

Volumn 111, Issue 2, 2014, Pages 212-213

  Jurecka, Agnieszka ^a,b   Malinova, Vera ^c   Tylki Szymańska, Anna ^a  

^a CHILDREN'S MEMORIAL HEALTH INSTITUTE   (Poland)

^b UNIVERSITY OF GDA SK   (Poland)

^c CHARLES UNIVERSITY   (Czech Republic)

Author keywords

Enzyme replacement therapy; Hunter syndrome; Maroteaux Lamy syndrome; Mucopolysaccharidosis type II; Mucopolysacchridosis type VI

Indexed keywords

GALSULFASE; IDURONATE 2 SULFATASE;

CATAPLEXY; COMPASSIONATE USE; CONJUNCTIVITIS; CZECH REPUBLIC; DISEASE COURSE; DISEASE SEVERITY; ENZYME REPLACEMENT; FATIGUE; HUMAN; HYDROCEPHALUS; HYPOTENSION; JOINT CONTRACTURE; JOINT STIFFNESS; LETTER; LITHUANIA; MAROTEAUX LAMY SYNDROME; NARCOLEPSY; PNEUMONIA; POLAND; PRIORITY JOURNAL; RECURRENT INFECTION; RESPIRATORY TRACT INFECTION; SINUSITIS; SPLENOMEGALY; TACHYCARDIA; THROMBOCYTE COUNT; THROMBOCYTOPENIA;

ENZYME REPLACEMENT THERAPY; HUNTER SYNDROME; MAROTEAUX-LAMY SYNDROME; MUCOPOLYSACCHARIDOSIS TYPE II; MUCOPOLYSACCHRIDOSIS TYPE VI;

ADOLESCENT; CHILD; DISEASE PROGRESSION; DRUG ADMINISTRATION SCHEDULE; ENZYME REPLACEMENT THERAPY; EUROPE, EASTERN; FEMALE; HUMANS; INJECTIONS, INTRAVENOUS; MALE; MUCOPOLYSACCHARIDOSES; N-ACETYLGALACTOSAMINE-4-SULFATASE; RECOMBINANT PROTEINS; WITHHOLDING TREATMENT;

EID: 84893706920     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2013.08.019     Document Type: Letter

References (3)

1. Jurecka A., Zuber Z., Opoka-Winiarska V., Wegrzyn G., Tylki-Szymanska A. Effect of rapid cessation of enzyme replacement therapy: a report of 5 cases and a review of the literature. Mol. Genet. Metab. 2012, 107:508-512.
2. Anbu A.T., Mercer J., Wraith J.E. Effect of discontinuing of laronidase in a patient with mucopolysaccharidosis type I. J. Inherit. Metab. Dis. 2006, 29:230-231.
3. Wegrzyn G., Tylki-Szymańska A., Liberek A., Piotrowska E., Jakóbkiewicz-Banecka J., Marucha J., Czartoryska B., Wegrzyn A. Rapid deterioration of a patient with mucopolysaccharidosis type I during interruption of enzyme replacement therapy. Am. J. Med. Genet. A 2007, 143A:1925-1927.