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 …

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 …

Spinal muscular atrophy (SMA) is a motor neuron disease. Nusinersen, a splice-switching
antisense oligonucleotide (ASO), was the first approved drug to treat SMA. Based on prior …

Spinal muscular atrophy (SMA) is an autosomal recessive disorder with an incidence of
1/6,000–1/10,000 and is the leading fatal disease among infants. Previously, there was no …

Until the recent development of disease-modifying therapeutics, spinal muscular atrophy
(SMA) was considered a devastating neuromuscular disease with a poor prognosis for most …

Abstract Spinal Muscular Atrophy results from loss-of-function mutations in SMN1 but
correcting aberrant splicing of SMN2 offers hope of a cure. However, current splice therapy …

Spinal muscular atrophy (SMA) is a neuromuscular genetic disease caused by reduced
survival motor neuron (SMN) protein. SMN is ubiquitous and deficient levels cause spinal …

Spinal muscular atrophy (SMA) is a fatal genetic disease, mainly affecting children. A
number of recent studies show, aside from lower motor neuron degeneration and atrophy of …

Accumulating evidence has revealed that survivin expression is associated with a malignant
phenotype and poor prognosis in glioma. Survivin is also a potential target of microRNA …

L-methioninase degrades methionine, which is essential in methionine-dependent cancer
cells, resulting in specific cell death. Normal cells can synthesize their own methionine …