Zobrazeno 1 - 1
of 1
pro vyhledávání: '"spinal muscular atrrophy"'
Autor:
R. Zanin, A. Seferian, N. Brolatti, A. Govoni, L. Edel, A. Jollet, M. Del Sole, Arseniy Lavrov, M.T. Arnoldi, E. De Vos, Eugenio Mercuri, L. Antonaci, G. Coratti, M. Pedemonte, Haojun Ouyang, K. Groves, O. Schneider, M. Foa, Volker Straub, A.C. Defeldre, G. Comi, Stefania Corti, V. Parente, A. Jonas, R.M. Lofra, Laurent Servais, Riccardo Masson, L. Buscemi, Nicolas Deconinck, S. De Lucia, Aurore Daron, M.C. Pera, Claudio Bruno, E. Pagliano, S. Mouffak, Nuno Mendonca, A. Vanlander, Deepa H. Chand, E. Thompson, H. Van Ruiten, V. Tahon, Giovanni Baranello, S. Tachibana, M. Pane, S. Morando, Francesco Muntoni, R. de Sanctis, V. Schembri, F. Dal Farra, A. Mandelli, Odile Boespflug-Tanguy, Sitra Tauscher-Wisniewski, M. Scoto, F. Abel, F. Magri
Publikováno v:
The Lancet Neurology. 20:832-841
Summary Background Spinal muscular atrophy is a rare, autosomal recessive, neuromuscular disease caused by biallelic loss of the survival motor neuron 1 (SMN1) gene, resulting in motor neuron dysfunction. In this STR1VE-EU study, we aimed to evaluate