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

Hereditary diseases are caused by mutations in genes, and more than 7,000 rare diseases
affect over 30 million Americans. For more than 30 years, hundreds of researchers have …

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 …

Neurodegenerative diseases, the leading cause of morbidity and disability, are gaining
increased attention as they impose a considerable socioeconomic impact, due in part to the …

Onasemnogene abeparvovec (onasemnogene abeparvovec-xioi; formerly AVXS-101;
ZOLGENSMA®) is an adeno-associated viral vector-based gene therapy designed to deliver …

Spinal muscular atrophy (SMA) is a rare and devastating disease. New disease-modifying
treatments have recently been approved and early treatment has been related to a better …

Spinal muscular atrophy (SMA) is one of the most common autosomal recessive diseases
with progressive weakness of skeletal and respiratory muscles, leading to significant …

Spinal muscular atrophy (SMA) is a neuromuscular disorder caused by loss or mutations in
SMN1. According to age of onset, achieved motor abilities, and life span, SMA patients are …

Abstract Spinal Muscular Atrophy (SMA) is caused by autosomal recessive mutations in
SMN1 and results in the loss of motor neurons and progressive muscle weakness. The …

Spinal muscular atrophy (SMA) is a devastating childhood motor neuron disease that, in the
most severe cases and when left untreated, leads to death within the first two years of life …