Adeno-associated viral (AAV) vectors are a rapidly emerging gene therapy platform for the
treatment of neurological diseases. In preclinical studies, transgenes encoding therapeutic …

Spinal muscular atrophy (SMA) is an autosomal recessive motor neuron disease caused by
deletion or mutation of SMN1. Four subtypes exist, characterized by different clinical …

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

Importance This ongoing study assesses long-term safety and durability of response in
infants with spinal muscular atrophy (SMA) type 1 after dosing with onasemnogene …

Spinal muscular atrophy (SMA), the leading genetic cause of infant mortality, arises from
survival motor neuron (SMN) protein insufficiency resulting from SMN1 loss. Approved …

Background Type 1 spinal muscular atrophy (SMA) is a progressive neuromuscular disease
characterized by an onset at 6 months of age or younger, an inability to sit without support …

Spinal muscular atrophy (SMA) is a neurodegenerative disease associated with severe
muscle atrophy and weakness in the limbs and trunk. We report interim efficacy and safety …

Background Spinal muscular atrophy is a rare, autosomal recessive, neuromuscular disease
caused by biallelic loss of the survival motor neuron 1 (SMN1) gene, resulting in motor …

Background Type 1 spinal muscular atrophy is a rare, progressive neuromuscular disease
that is caused by low levels of functional survival of motor neuron (SMN) protein. Risdiplam …

Background Spinal muscular atrophy type 1 is a motor neuron disorder resulting in death or
the need for permanent ventilation by age 2 years. We aimed to evaluate the safety and …