Titre : | A series of case reports from JEWELFISH, an open-label study to investigate the safety, tolerability, and pharmacokinetics/ pharmacodynamics of RG7916 in adult and pediatric patients with spinal muscular atrophy who previously participated in a study with another SMN2-targeting therapy |
Revue : | Neuromuscular disorders : NMD, 27, s2 |
Auteurs : | Chiriboga C ; Mercuri E ; Fischer D ; Marquet A ; Kraus D ; Alexander M ; Cho A ; Armstrong G ; Kletzl H ; Czech C ; Seabrook T ; Abdallah H ; Mueller L ; Gorni K ; Khwaja O |
Type de document : | Article |
Année de publication : | 2017 |
Pages : | S209 |
Langues: | Anglais |
Résumé : |
Poster présenté lors :
22nd International Congress of the World Muscle Society 3-7 October 2017 • St Malo, France Volume 27, Supplement 2, Pages S51-S270 (October 2017) |
Pubmed / DOI : | DOI : 10.1016/j.nmd.2017.06.416 |