Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy
Sign Up to like & getrecommendations! Published in 2022 at "Annals of Clinical and Translational Neurology"
DOI: 10.1002/acn3.51519
Abstract: To provide a greater understanding of the tolerability, safety and clinical outcomes of onasemnogene abeparvovec in real‐world practice, in a broad population of infants with spinal muscular atrophy (SMA). read more here.
Keywords: safety; muscular atrophy; abeparvovec spinal; onasemnogene abeparvovec ... See more keywords
Thérapie génique (TG) dans l’amyotrophie spinale de type 1 (SMA1) : suivi à long terme (SLT) de l’essai clinique de phase 1 évaluant l’onasemnogène abeparvovec
Sign Up to like & getrecommendations! Published in 2020 at "Revue neurologique"
DOI: 10.1016/j.neurol.2020.01.111
Abstract: Introduction L’onasemnogene abeparvovec (AVXS-101), une TG a administration unique par voie intraveineuse qui traite la cause genetique principale de la SMA. Objectifs Evaluer la securite et l’efficacite a long terme de l’onasemnogene abeparvovec a forte… read more here.
Keywords: dans etude; onasemnogene abeparvovec; abeparvovec; long terme ... See more keywords
Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue.
Sign Up to like & getrecommendations! Published in 2021 at "Nature medicine"
DOI: 10.1038/s41591-021-01483-7
Abstract: Spinal muscular atrophy type 1 (SMA1) is a debilitating neurodegenerative disease resulting from survival motor neuron 1 gene (SMN1) deletion/mutation. Onasemnogene abeparvovec (formerly AVXS-101) is a gene therapy that restores SMN production via one-time systemic… read more here.
Keywords: abeparvovec dna; administration; biodistribution; smn protein ... See more keywords
Onasemnogene abeparvovec for the treatment of spinal muscular atrophy
Sign Up to like & getrecommendations! Published in 2022 at "Expert Opinion on Biological Therapy"
DOI: 10.1080/14712598.2022.2066471
Abstract: ABSTRACT Introduction Gene therapy for spinal muscular atrophy (SMA) represents a significant milestone in the treatment of neurologic diseases. SMA is a neurodegenerative disease that results in motor neuron loss because of mutations of the… read more here.
Keywords: treatment; gene; onasemnogene abeparvovec; gene therapy ... See more keywords
Single-Dose Intrathecal Dorsal Root Ganglia Toxicity of Onasemnogene Abeparvovec in Cynomolgus Monkeys
Sign Up to like & getrecommendations! Published in 2022 at "Human Gene Therapy"
DOI: 10.1089/hum.2021.255
Abstract: Intravenous onasemnogene abeparvovec is approved for the treatment of spinal muscular atrophy in children read more here.
Keywords: root; onasemnogene abeparvovec; toxicity; onasemnogene ... See more keywords
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
G32 Improving survival of those with cerebral palsy in England and Wales over the last 18 years
Sign Up to like & getrecommendations! Published in 2020 at "Archives of Disease in Childhood"
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
Child Neurology: Pathologically Confirmed Thrombotic Microangiopathy Caused by Onasemnogene Abeparvovec Treatment for SMA
Sign Up to like & getrecommendations! Published in 2022 at "Neurology"
DOI: 10.1212/wnl.0000000000200676
Abstract: Onasemnogene abeparvovec is an adeno-associated virus vector-based gene therapy for spinal muscular atrophy (SMA). Although several cases of drug-induced thrombotic microangiopathy due to onasemnogene abeparvovec have been reported, none has been confirmed pathologically. Here, we… read more here.
Keywords: thrombotic microangiopathy; neurology; abeparvovec treatment; microscopy ... See more keywords
Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec Treatment.
Sign Up to like & getrecommendations! Published in 2023 at "Journal of neuromuscular diseases"
DOI: 10.3233/jnd-221531
Abstract: BACKGROUND Improvement and maintenance of bulbar function are goals of disease-modifying treatments for spinal muscular atrophy (SMA). Lack of standardized measures and a widely accepted definition of bulbar function represents a gap in SMA care.… read more here.
Keywords: spinal muscular; bulbar; onasemnogene abeparvovec; muscular atrophy ... See more keywords
Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG)
Sign Up to like & getrecommendations! Published in 2023 at "Journal of Neuromuscular Diseases"
DOI: 10.3233/jnd-221560
Abstract: Background: Spinal muscular atrophy (SMA) is a neuromuscular disorder arising from biallelic non-functional survival motor neuron 1 (SMN1) genes with variable copies of partially functional SMN2 gene. Intrathecal onasemnogene abeparvovec administration, at fixed, low doses,… read more here.
Keywords: intrathecal onasemnogene; sitting nonambulatory; dose; onasemnogene abeparvovec ... See more keywords