Mapping the differences in care for 5,000 Spinal Muscular Atrophy patients, a survey of 24 national registries in North America, Australasia and Europe

Author(s):
Bladen, C.L.; Thompson, R.; Jackson, J.M.; Garland, C.; Wegel, C.; Ambrosini, A.; Pisano, P.; Walter, M.C.; Schreiber, O.; Lusakowska, A.; Jedrzejowska, M.; Kostera-Pruszczyk, A.; Pol, L. van der; Wadman, R.I.; Gredal, O.; Karaduman, A.; Topaloglu, H.; Yilmaz, O.; Matyushenko, V.; Rasic, V.M.; Kosac, A.; Karcagi, V.; Garami, M.; Herczegfalvi, A.; Monges, S.; Moresco, A.; Chertkoff, L.; Chamova, T.; Guergueltcheva, V.; Butoianu, N.; Craiu, D.; Korngut, L.; Campbell, C.; Haberlova, J.; Strenkova, J.; Alejandro, M.; Jimenez, A.; Ortiz, G.G.; Enriquez, G.V.G.; Rodrigues, M.; Roxburgh, R.; Dawkins, H.; Youngs, L.; Lahdetie, J.; Angelkova, N.; Saugier-Veber, P.; Cuisset, J.M.; Bloetzer, C.; Jeannet, P.Y.; Klein, A.; Nascimento, A.; Tizzano, E.; Salgado, D.; Mercuri, E.; Sejersen, T.; Kirschner, J.; Rafferty, K.; Straub, V.; Bushby, K.; Verschuuren, J.; Beroud, C.; Lochmuller, H.
Published
2014
Publication type
Article / Letter to editor
Spinal muscular atrophyRare diseaseDisease registriesTREAT-NMD
Date issued
2014-01-31
Language
English
Is part of
Journal of Neurology (2014), Vol. 261, no. 1, p. 152-163
hdl
1887/107039
doi
10.1007/s00415-013-7154-1