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 …

Protein–RNA interactions have crucial roles in various cellular activities, which, when
dysregulated, can lead to a range of human diseases. The identification of small molecules …

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 congenital neuromuscular disorder characterized by
motor neuron loss, resulting in progressive weakness. SMA is notable in the health care …

Spinal muscular atrophy is one of the most common neuromuscular disorders of childhood
and has high morbidity and mortality. Three different disease-modifying treatments were …

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 …

RNA binding proteins (RBPs) are a large and diverse class of proteins that regulate all
aspects of RNA biology. As RBP dysregulation has been implicated in a number of human …

Huntington's disease (HD) is a progressive neurological disease that is inherited in an
autosomal fashion. The cause of disease pathology is an expansion of cytosine-adenine …

Alternative splicing (AS) serves as a pivotal mechanism in transcriptional regulation,
engendering transcript diversity, and modifications in protein structure and functionality …

The growing information currently available on the central role of non-coding RNAs
(ncRNAs) including microRNAs (miRNAS) and long non-coding RNAs (lncRNAs) for chronic …