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

Many diseases are caused by insufficient expression of mutated genes and would benefit
from increased expression of the corresponding protein. However, in drug development, it …

Designing an RNA-interacting molecule that displays high therapeutic efficacy while
retaining specificity within a broad concentration range remains a challenging task …

Spinal muscular atrophy (SMA) is caused by mutations in SMN1. SMN2 is a paralogous
gene with a C• G-to-T• A transition in exon 7, which causes this exon to be skipped in most …

Introduction Gene therapy for spinal muscular atrophy (SMA) represents a significant
milestone in the treatment of neurologic diseases. SMA is a neurodegenerative disease that …

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease caused
by deletion or mutation of the SMN1 gene. It is characterized by a progressive loss of motor …

Spinal deformities are considered an important complication of neuromuscular disorders
such as spinal muscular atrophy (SMA). SMA patients typically develop progressive early …

Spinal muscular atrophy (SMA) is a devastating neuromuscular disease caused by
mutations in the SMN1 gene. Despite the development of various therapies, outcomes can …

Proximal spinal muscular atrophy (SMA) is a leading genetic cause for infant death in the
world and results from the selective loss of motor neurons in the spinal cord. SMA is a …

Onasemnogene abeparvovec is a recombinant adeno-associated virus serotype 9 (AAV9)
vector-based gene therapy for spinal muscular atrophy (SMA). Patients with elevated titers of …