Survival, Motor Function, and Motor Milestones: Comparison of AVXS-101 Relative to Nusinersen for the Treatment of Infants with Spinal Muscular Atrophy Type 1

INTRODUCTION: Infants with spinal muscular atrophy (SMA) type 1 typically face a decline in motor function and a severely shortened life expectancy. Clinical trials for SMA type 1 therapies, onasemnogene abeparvovec (AVXS-101) and nusinersen, demonstrated meaningful improvements in efficacy (e.g., o...

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Publicat a:Adv Ther
Autors principals: Dabbous, Omar, Maru, Benit, Jansen, Jeroen P., Lorenzi, Maria, Cloutier, Martin, Guérin, Annie, Pivneva, Irina, Wu, Eric Q., Arjunji, Ramesh, Feltner, Douglas, Sproule, Douglas M.
Format: Artigo
Idioma:Inglês
Publicat: Springer Healthcare 2019
Matèries:
Original Research
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC6824368/
https://ncbi.nlm.nih.gov/pubmed/30879249
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s12325-019-00923-8
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