The TREAT-NMD Duchenne Muscular Dystrophy Registries: Conception, Design, and Utilization by Industry and Academia

Autor: Bladen, Catherine, Rafferty, Karen, Straub, Volker, Monges, Soledad, Moresco, Angélica, Dawkins, Hugh, Roy, Anna, Chamova, Teodora, Guergueltcheva, Velina, Korngut, Lawrence, Campbell, Craig, Dai, Yi, Barišić, Nina, Kos, Tea, Brabec, Petr, Rahbek, Jes, Lahdetie, Jaana, Tuffery-Giraud, Sylvie, Claustres, Mireille, Leturcq, France, Ben Yaou, Rabah, Walter, Maggie C, Schreiber, Olivia, Karcagi, Veronika, Herczegfalvi, Agnes, Viswanathan, Venkatarman, Bayat, Farhad, de La Caridad Guerrero Sarmiento, Isis, Ambrosini, Anna, Ceradini, Francesc, Kimura, En, van den Bergen, Jan, Rodrigues, Miriam, Roxburgh, Richard, Lusakowska, Anna, Oliveira, Jorge, Santos, Rosário, Neagu, Elena, Butoianu, Niculina, Artemieva, Svetlana, Rasic, Vedrana Milic, Posada, Manuel, Palau, Francesc, Lindvall, Björn, Bloetzer, Clemens, Karaduman, Ayşe, Topaloğlu, Haluk, Inal, Serap, Oflazer, Piraye, Stringer, Angela, Shatillo, Andriy V, Martin, Ann S, Peay, Holly, Flanigan, Kevin M, Salgado, David, von Rekowski, Brigitta, Lynn, Stephen, Heslop, Emma, Gainotti, Sabina, Taruscio, Domenica, Kirschner, Jan, Verschuuren, Jan, Bushby, Kate, Béroud, Christophe, Lochmuller, Hanns, Roy, Hugh, Tuffery-Giraud, France, Claustres, France, Walter, Maggie C., Shatillo, Andriy V., Martin, Ann S., Flanigan, Kevin M.

Publikováno v: Human Mutation, 34(11), 1449-1457
HUMAN MUTATION
r-IIS La Fe. Repositorio Institucional de Producción Científica del Instituto de Investigación Sanitaria La Fe
instname
Human Mutation
Human Mutation, 2013, 34 (11), pp.1449-1457. ⟨10.1002/humu.22390⟩
Human Mutation, Wiley, 2013, 34 (11), pp.1449-1457. ⟨10.1002/humu.22390⟩

International audience; Duchenne muscular dystrophy (DMD) is an X-linked genetic disease, caused by the absence of the dystrophin protein. Although many novel therapies are under development for DMD, there is currently no cure and affected individual

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::6c2647260e6ae2f3061809a1dc3d0113
http://hdl.handle.net/1887/100815