Proximal spinal muscular atrophy (SMA), a leading genetic cause of infant death worldwide,
is an early-onset, autosomal recessive neurodegenerative disease characterized by the loss …

Spinal muscular atrophy (SMA) is a leading genetic cause of infant death worldwide that is
characterized by loss of spinal motor neurons leading to muscle weakness and atrophy …

Fragile X messenger ribonucleoprotein 1 protein (FMRP) binds many mRNA targets in the
brain. The contribution of these targets to fragile X syndrome (FXS) and related autism …

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 …

Recent unprecedented advances in treatment for spinal muscular atrophy (SMA) enabled
patients to access the first approved disease modifying therapy for the condition. There are …

Spinal muscular atrophy (SMA) is currently classified into five different subtypes, from the
most severe (type 0) to the mildest (type 4) depending on age at onset, best motor function …

After 26 years of discovery of the determinant survival motor neuron 1 and the modifier
survival motor neuron 2 genes (SMN1 and SMN2, respectively), three SMN-dependent …

Spinal Muscular Atrophy (SMA) is a neuromuscular disorder caused by mutations in the
SMN1 gene. Being a monogenic disease, it is characterized by high clinical heterogeneity …

Abstract Background/Purpose Spinal muscular atrophy (SMA) is an autosomal recessive
neuromuscular disorder, considered one of the leading causes of infant mortality. It is …

Introduction Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular
disorder caused by the degeneration of motor neurons, muscle weakness, and atrophy that …