Onasemnogene abeparvovec in type 1 spinal muscular atrophy: A systematic review and meta-analysis.
Sign Up to like & getrecommendations! Published in 2022 at "Human gene therapy"
DOI: 10.1089/hum.2022.161
Abstract: One of the latest approved therapies for spinal muscular atrophy (SMA) is onasemnogene abeparvovec, which transduces motor neurons with the survival of motor neuron gene. The aim of this meta-analysis was to estimate the effect… read more here.
Keywords: meta analysis; onasemnogene abeparvovec; systematic review; motor ... See more keywords
Nusinersen treatment of Spinal Muscular Atrophy Type 1 - results of expanded access programme in Poland.
Sign Up to like & getrecommendations! Published in 2021 at "Neurologia i neurochirurgia polska"
DOI: 10.5603/pjnns.a2021.0020
Abstract: AIM OF THE STUDY This study aimed to evaluate the effects of nusinersen therapy in Polish children with SMA type 1. Clinical rationale of study. Spinal muscular atrophy (SMA) is a neuromuscular disorder that is… read more here.
Keywords: atrophy; treatment; sma type; nusinersen treatment ... See more keywords