Dados do Trabalho
Título
SUNFISH Parts 1 and 2: 4-year efficacy and safety data of risdiplam in Types 2 and 3 SMA
Palavras-chave
spinal muscular atrophy, risdiplam, efficacy
Área
Doenças Neuromusculares
Autores
Alexandra PQC Araujo, Maryam Oskoui, John W Day, Nicolas Deconinck, Elena S Mazzone, Andres Nasimento, Kayoko Saito, Carole Vuillerot, Giovanni Baranello, Odile Boespflug-Tanguy, Nathalie Goemans, Janbernd Kirschner, Anna Kostera-Pruszczyk, Laurent Servais, Jessica Braid, Marianne Gerber, Ksenija Gorni, Carmen Martin, Wai Yin Yeung, Renata S Scalco, Eugenio Mercuri