Spinal muscular atrophy (SMA) is the most common genetically inherited neurodegenerative
disease resulting in infant mortality. SMA is caused by genetic deletion or mutation in the …

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 …

Paucity of the survival motor neuron (SMN) protein triggers the oft-fatal infantile-onset motor
neuron disorder, spinal muscular atrophy (SMA). Augmenting the protein is one means of …

Objective Spinal muscular atrophy (SMA) is an inherited neuromuscular disorder leading to
paralysis and subsequent death in young children. Initially considered a motor neuron …

Importance The important depletion of mitochondrial DNA (mtDNA) and the general
depression of mitochondrial respiratory chain complex levels (including complex II) have …

Facioscapulohumeral muscular dystrophy (FSHD), a common hereditary myopathy, is
caused either by the contraction of the D4Z4 macrosatellite repeat at the distal end of …

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 an autosomal recessive neurodegenerative disease
characterized by the selective death of lower motor neurons in the brain stem and spinal …

Spinal muscular atrophy (SMA) is a genetic neurological disease that causes infant
mortality; no effective therapies are currently available. SMA is due to homozygous …

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