Spinal muscular atrophy (SMA) is one of the leading causes of infant mortality. SMA is
mostly caused by low levels of Survival Motor Neuron (SMN) protein due to deletion of or …

Spinal muscular atrophy is typically characterized as a motor neuron disease. Untreated
patients with the most severe form, spinal muscular atrophy type 1, die early with infantile …

Background & Aims Spinal muscular atrophy (SMA) is an autosomal recessive, childhood-
onset motor neuron disease. Onasemnogene abeparvovec (OA) is a gene therapy designed …

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 …

Spinal muscular atrophy is a neurodegenerative disease resulting from irreversible loss of
anterior horn cells owing to biallelic deletions/mutations in the survival motor neuron (SMN) …

Loss or deletion of survival motor neuron 1 gene (SMN1) is causative for a severe and
devastating neuromuscular disease, Spinal Muscular Atrophy (SMA). SMN1 produces SMN …

Spinal muscular atrophy (SMA) is an autosomal recessive, neurodegenerative disease
caused by biallelic mutations in the survival motor neuron 1 (SMN1) gene. SMA is …

Spinal muscular atrophy (SMA) is a devastating neuromuscular disorder caused by
recessive mutations in the SMN1 gene, globally affecting~ 8–14 newborns per 100,000. The …

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 …

Spinal Muscular Atrophy (SMA) is monogenic motoneuron disease caused by low levels of
the Survival of Motoneuron protein (SMN). Recently, two different drugs were approved for …