peer reviewedINTRODUCTION: Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative neuromuscular disease that presents primarily in children. Abnormalities in the SMN1 gene cause reduced levels of the survival motor neuron (SMN) protein, while a second gene, SMN2, produces low levels of functional SMN protein. Currently available drugs do not cure, so a significant unmet need remains for patients treated after symptom onset. AREAS COVERED: Drugs available in the clinic, investigational agents and key questions for researchers are discussed. A pragmatic search of the literature was performed to identify therapies in late stages of preclinical, or in early stages of clinical development. This list was compared to the CureSM...
Spinal muscular atrophy (SMA) is a progressive, recessively inherited neuromuscular disease, charact...
BACKGROUND: Spinal muscular atrophy (SMA) is caused by a homozygous deletion of the survival motor n...
Introduction: Currently, there is no cure available for the hereditary neurodegenerative disease pro...
peer reviewed[en] INTRODUCTION: Spinal muscular atrophy (SMA) is an autosomal recessive neurodegener...
Spinal muscular atrophy (SMA) is a childhood disorder caused by loss of the survival motor neuron (S...
Until the recent development of disease-modifying therapeutics, spinal muscular atrophy (SMA) was co...
Spinal muscular atrophy (SMA) is one of the leading causes of infant mortality. SMA is mostly caused...
Spinal muscular atrophy (SMA) is a devastating neuromuscular disorder characterized by loss of motor...
Introduction: Spinal muscular atrophy (SMA) is one of the most common genetically determined causes ...
Spinal muscular atrophy (SMA) is a progressive neurodegenerative disease with an autosomal recessive...
Spinal muscular atrophy (SMA) is a devastating neuromuscular disorder characterized by loss of motor...
Background Spinal muscular atrophy (SMA) is an autosomal recessive motor neuron disease caused by mu...
Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disease resulting from pat...
AbstractHumans have two nearly identical copies of survival motor neuron gene: SMN1 and SMN2. Deleti...
Spinal Muscular Atrophy (SMA) is uncommon genetic (autosomal recessive) disease that deteriorates ne...
Spinal muscular atrophy (SMA) is a progressive, recessively inherited neuromuscular disease, charact...
BACKGROUND: Spinal muscular atrophy (SMA) is caused by a homozygous deletion of the survival motor n...
Introduction: Currently, there is no cure available for the hereditary neurodegenerative disease pro...
peer reviewed[en] INTRODUCTION: Spinal muscular atrophy (SMA) is an autosomal recessive neurodegener...
Spinal muscular atrophy (SMA) is a childhood disorder caused by loss of the survival motor neuron (S...
Until the recent development of disease-modifying therapeutics, spinal muscular atrophy (SMA) was co...
Spinal muscular atrophy (SMA) is one of the leading causes of infant mortality. SMA is mostly caused...
Spinal muscular atrophy (SMA) is a devastating neuromuscular disorder characterized by loss of motor...
Introduction: Spinal muscular atrophy (SMA) is one of the most common genetically determined causes ...
Spinal muscular atrophy (SMA) is a progressive neurodegenerative disease with an autosomal recessive...
Spinal muscular atrophy (SMA) is a devastating neuromuscular disorder characterized by loss of motor...
Background Spinal muscular atrophy (SMA) is an autosomal recessive motor neuron disease caused by mu...
Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disease resulting from pat...
AbstractHumans have two nearly identical copies of survival motor neuron gene: SMN1 and SMN2. Deleti...
Spinal Muscular Atrophy (SMA) is uncommon genetic (autosomal recessive) disease that deteriorates ne...
Spinal muscular atrophy (SMA) is a progressive, recessively inherited neuromuscular disease, charact...
BACKGROUND: Spinal muscular atrophy (SMA) is caused by a homozygous deletion of the survival motor n...
Introduction: Currently, there is no cure available for the hereditary neurodegenerative disease pro...