Spinal muscular atrophy (SMA) is a neurodegenerative disorder caused by mutations in
SMN1 (encoding survival motor neuron protein (SMN)). Reduced expression of SMN leads …

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 …

Abstract SPR1NT (NCT03505099) was a Phase III, multicenter, single-arm study to
investigate the efficacy and safety of onasemnogene abeparvovec for presymptomatic …

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 …

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 …

Onasemnogene abeparvovec (Zolgensma®) is a gene therapy approved for the treatment of
spinal muscular atrophy (SMA). Administered as a one-time intravenous infusion …

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 …

Spinal muscular atrophy (SMA) is a genetically inherited recessive neuromuscular disease
that causes muscular atrophy and weakness. Onasemnogene abeparvovec (formerly AVXS …

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 …

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 …