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pro vyhledávání: '"Bonnie Bielefeld"'
Publikováno v:
Molecular Genetics and Metabolism. 110:303-310
In the pivotal phase II/III trial of idursulfase administered intravenously to treat mucopolysaccharidosis II, approximately half of the patients developed antibodies to idursulfase. This post-hoc analysis of data from the phase II/III trial and exte
Publikováno v:
Orphanet Journal of Rare Diseases
Background Twenty-eight treatment-naïve mucopolysaccharidosis II patients (16 months–7.5 years) received 0.5 mg/kg idursulfase weekly for one year in NCT00607386. Serum anti-idursulfase immunoglobulin G antibodies (Abs) were seen in 68% of patient
Autor:
Christian J. Hendriksz, Ann J. Barbier, Yune Kunes, Ashok Vellodi, Bonnie Bielefeld, Roberto Giugliani, Suresh Vijayaraghavan, Paul Harmatz, Kenneth Sciarappa, Arian Pano, Nancy J. Mendelsohn
Publikováno v:
Molecular Genetics and Metabolism. 111:S53
38% by 240 weeks (n= 5). Forced expiratory volume (FEV1) increased approximately 11% at 96 weeks (n = 33) and was maintained at 240 weeks (n= 5) (3). After 7 years, FVC improved by 47% (n= 5) and FEV1 by 34% (n= 6), showing continued upward trajector