The overproduction of reactive oxygen species (ROS) and consequent oxidative stress
contribute to the pathogenesis of acute and chronic liver diseases. It is now acknowledged …

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 …

Antisense oligonucleotides (ASOs) have emerged as one of the most innovative new
genetic drug modalities. However, their high molecular weight limits their bioavailability for …

Spinal muscular atrophy (SMA) is an autosomal recessive genetic disorder leading to
paralysis, muscle atrophy, and death. Significant advances in antisense oligonucleotide …

Spinal muscular atrophy (SMA) is typically characterized as a motor neuron disease, but
extraneuronal phenotypes are present in almost every organ in severely affected patients …

Background: With the increasing prevalence of obesity and metabolic syndrome, the
incidence of non-alcoholic fatty liver disease (NAFLD) is also increasing. In the next decade …

Background The liver is a key metabolic organ, acting as a hub to metabolically connect
various tissues. Spinal muscular atrophy (SMA) is a neuromuscular disorder whereby …

Abstract Background and Objective Spinal muscular atrophy (SMA) is a genetic disorder
with limited treatment options. It is crucial to have a comprehensive understanding of drug …

Spinal muscular atrophy (SMA) is a monogenic neuromuscular disease caused by low
levels of the Survival Motor Neuron (SMN) protein. Motor neuron degeneration is the central …

Spinal muscular atrophy (SMA) is an inherited neuromuscular disorder stemming from
deletions or mutations in the Survival Motor Neuron 1 (SMN1) gene, leading to decreased …