Long term survival and cardiopulmonary outcome in children with Hurler syndrome after haematopoietic stem cell transplantation
Author:
Publisher
Wiley
Subject
Genetics (clinical),Genetics
Link
http://link.springer.com/content/pdf/10.1007/s10545-017-0034-6.pdf
Reference19 articles.
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2. Bigger BW, Wynn RF (2014) Novel approaches and mechanisms in hematopoietic stem cell gene therapy. Discov Med 17(94):207–215
3. Bijarnia S, Shaw P, Vimpani A, Smith R, Pacey V, O’Grady H, Christodoulou J, Sillence D (2009) Combined enzyme replacement and haematopoietic stem cell transplantation in Hurler syndrome. J Paediatr Child Health 45(7-8):469–472
4. Boelens JJ, Wynn RF, O’Meara A, Veys P, Bertrand Y, Souillet G, Wraith JE, Fischer A, Cavazzana-Calvo M, Sykora KW, Sedlacek P, Rovelli A, Uiterwaal CS, Wulffraat N (2007) Outcomes of hematopoietic stem cell transplantation for Hurler’s syndrome in Europe: a risk factor analysis for graft failure. Bone Marrow Transplant 40(3):225–233
5. Boelens JJ, Rocha V, Aldenhoven M, Wynn R, O’Meara A, Michel G, Ionescu I, Parikh S, Prasad VK, Szabolcs P, Escolar M, Gluckman E, Cavazzana-Calvo M, Kurtzberg J, I. e. W. P. o. E. Eurocord and U. Duke (2009) Risk factor analysis of outcomes after unrelated cord blood transplantation in patients with hurler syndrome. Biol Blood Marrow Transplant 15(5):618–625
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