G32 Improving survival of those with cerebral palsy in England and Wales over the last 18 years
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DOI: 10.1136/archdischild-2020-rcpch.21
Abstract: Aims Report onasemnogene abeparvovec (formerly AVXS101) GRT development for SMA. Onasemnogene abeparvovec is a one-time, intravenous GRT that addresses the genetic root cause of SMA, a progressive neurological disease. Onasemnogene abeparvovec delivers the survival motor… read more here.
Keywords: improving survival; g32 improving; onasemnogene abeparvovec; cohort ... See more keywords