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Usefulness of bone marrow transplantation in the Hurler syndrome.
Braunlin EA, Stauffer NR, Peters CH, Bass JL, Berry JM, Hopwood JJ, Krivit W. Braunlin EA, et al. Among authors: hopwood jj. Am J Cardiol. 2003 Oct 1;92(7):882-6. doi: 10.1016/s0002-9149(03)00909-3. Am J Cardiol. 2003. PMID: 14516901 Clinical Trial.
Cord-blood transplants from unrelated donors in patients with Hurler's syndrome.
Staba SL, Escolar ML, Poe M, Kim Y, Martin PL, Szabolcs P, Allison-Thacker J, Wood S, Wenger DA, Rubinstein P, Hopwood JJ, Krivit W, Kurtzberg J. Staba SL, et al. Among authors: hopwood jj. N Engl J Med. 2004 May 6;350(19):1960-9. doi: 10.1056/NEJMoa032613. N Engl J Med. 2004. PMID: 15128896 Free article.
Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme®) therapy.
Braunlin E, Rosenfeld H, Kampmann C, Johnson J, Beck M, Giugliani R, Guffon N, Ketteridge D, Sá Miranda CM, Scarpa M, Schwartz IV, Leão Teles E, Wraith JE, Barrios P, Dias da Silva E, Kurio G, Richardson M, Gildengorin G, Hopwood JJ, Imperiale M, Schatz A, Decker C, Harmatz P; MPS VI Study Group. Braunlin E, et al. Among authors: hopwood jj. J Inherit Metab Dis. 2013 Mar;36(2):385-94. doi: 10.1007/s10545-012-9481-2. Epub 2012 Jun 5. J Inherit Metab Dis. 2013. PMID: 22669363 Free PMC article.
Towards gene therapy of Hurler syndrome.
Fairbairn LJ, Lashford LS, Spooncer E, McDermott RH, Lebens G, Arrand JE, Arrand JR, Bellantuono I, Holt R, Hatton CE, Cooper A, Besley GT, Wraith JE, Anson DS, Hopwood JJ, Dexter TM. Fairbairn LJ, et al. Among authors: hopwood jj. Cas Lek Cesk. 1997 Jan 8;136(1):27-31. Cas Lek Cesk. 1997. PMID: 9127508
Long-term in vitro correction of alpha-L-iduronidase deficiency (Hurler syndrome) in human bone marrow.
Fairbairn LJ, Lashford LS, Spooncer E, McDermott RH, Lebens G, Arrand JE, Arrand JR, Bellantuono I, Holt R, Hatton CE, Cooper A, Besley GT, Wraith JE, Anson DS, Hopwood JJ, Dexter TM. Fairbairn LJ, et al. Among authors: hopwood jj. Proc Natl Acad Sci U S A. 1996 Mar 5;93(5):2025-30. doi: 10.1073/pnas.93.5.2025. Proc Natl Acad Sci U S A. 1996. PMID: 8700879 Free PMC article.
392 results