Stability is maintained in adults with Gaucher disease type 1 switched from velaglucerase alfa to eliglustat or imiglucerase: A sub-analysis of the eliglustat ENCORE trial

por Pleat, Rebecca, Cox, Timothy M., Burrow, T. Andrew, Giraldo, Pilar, Goker-Alpan, Ozlem, Rosenbloom, Barry E., Croal, Laura R., Underhill, Lisa H., Gaemers, Sebastiaan J.M., Peterschmitt, M. Judith
Publicado 2016

Early treatment with laronidase improves clinical outcomes in patients with attenuated MPS I: a retrospective case series analysis of nine sibships

por Al-Sannaa, Nouriya A., Bay, Luisa, Barbouth, Deborah S., Benhayoun, Youssef, Goizet, Cyril, Guelbert, Norberto, Jones, Simon A., Kyosen, Sandra Obikawa, Martins, Ana Maria, Phornphutkul, Chanika, Reig, Celia, Pleat, Rebecca, Fallet, Shari, Ivanovska Holder, Iva
Publicado 2015