[HTML][HTML] AAV vectors applied to the treatment of CNS disorders: Clinical status and challenges
L Kang, S Jin, J Wang, Z Lv, C Xin, C Tan… - Journal of Controlled …, 2023 - Elsevier
In recent years, adeno-associated virus (AAV) has become the most important vector for
central nervous system (CNS) gene therapy. AAV has already shown promising results in …
central nervous system (CNS) gene therapy. AAV has already shown promising results in …
Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial
KA Strauss, MA Farrar, F Muntoni, K Saito… - Nature medicine, 2022 - nature.com
Abstract SPR1NT (NCT03505099) was a Phase III, multicenter, single-arm study to
investigate the efficacy and safety of onasemnogene abeparvovec for presymptomatic …
investigate the efficacy and safety of onasemnogene abeparvovec for presymptomatic …
Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial
KA Strauss, MA Farrar, F Muntoni, K Saito… - Nature medicine, 2022 - nature.com
Most children with biallelic SMN1 deletions and three SMN2 copies develop spinal muscular
atrophy (SMA) type 2. SPR1NT (NCT03505099), a Phase III, multicenter, single-arm trial …
atrophy (SMA) type 2. SPR1NT (NCT03505099), a Phase III, multicenter, single-arm trial …
Adeno-associated virus as a delivery vector for gene therapy of human diseases
JH Wang, DJ Gessler, W Zhan, TL Gallagher… - Signal Transduction and …, 2024 - nature.com
Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene
therapy owing to its minimal pathogenicity and ability to establish long-term gene expression …
therapy owing to its minimal pathogenicity and ability to establish long-term gene expression …
Onasemnogene Abeparvovec: A review in spinal muscular atrophy
HA Blair - Cns Drugs, 2022 - Springer
Onasemnogene abeparvovec (Zolgensma®) is a gene therapy approved for the treatment of
spinal muscular atrophy (SMA). Administered as a one-time intravenous infusion …
spinal muscular atrophy (SMA). Administered as a one-time intravenous infusion …
Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy
AM D'Silva, S Holland, D Kariyawasam… - Annals of clinical …, 2022 - Wiley Online Library
Objective 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 …
of onasemnogene abeparvovec in real‐world practice, in a broad population of infants with …
Onasemnogene abeparvovec gene replacement therapy for the treatment of spinal muscular atrophy: a real-world observational study
I Bitetti, V Lanzara, G Margiotta, A Varone - Gene Therapy, 2023 - nature.com
Spinal muscular atrophy (SMA) is a genetically inherited recessive neuromuscular disease
that causes muscular atrophy and weakness. Onasemnogene abeparvovec (formerly AVXS …
that causes muscular atrophy and weakness. Onasemnogene abeparvovec (formerly AVXS …
Onasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in naïve patients with spinal muscular atrophy and following switch from …
Background Efficacy and safety of onasemnogene abeparvovec (OA) for Spinal Muscular
Atrophy infants under 7 months and< 8.5 kg has been reported in clinical trials. This study …
Atrophy infants under 7 months and< 8.5 kg has been reported in clinical trials. This study …
New therapies for spinal muscular atrophy: where we stand and what is next
L Antonaci, MC Pera, E Mercuri - European Journal of Pediatrics, 2023 - Springer
The natural history of spinal muscular atrophy has been radically changed by the advent of
improved standards of care and the availability of disease-modifying therapies. The aim of …
improved standards of care and the availability of disease-modifying therapies. The aim of …