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Pharmacokinetic profile of recombinant human N-acetylgalactosamine 4-sulphatase enzyme replacement therapy in patients with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): a phase I/II study.
Harmatz P, Kramer WG, Hopwood JJ, Simon J, Butensky E, Swiedler SJ; Mucopolysaccharidosis VI Study Group. Harmatz P, et al. Among authors: kramer wg. Acta Paediatr Suppl. 2005 Mar;94(447):61-8; discussion 57. doi: 10.1111/j.1651-2227.2005.tb02115.x. Acta Paediatr Suppl. 2005. PMID: 15895715 Clinical Trial.
A phase 1 dose-escalation study: safety, tolerability, and pharmacokinetics of FBS0701, a novel oral iron chelator for the treatment of transfusional iron overload.
Rienhoff HY Jr, Viprakasit V, Tay L, Harmatz P, Vichinsky E, Chirnomas D, Kwiatkowski JL, Tapper A, Kramer W, Porter JB, Neufeld EJ. Rienhoff HY Jr, et al. Haematologica. 2011 Apr;96(4):521-5. doi: 10.3324/haematol.2010.034405. Epub 2010 Dec 20. Haematologica. 2011. PMID: 21173101 Free PMC article. Clinical Trial.
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