Objective Recent advances in understanding Spinal Muscular Atrophy (SMA)
etiopathogenesis prompted development of potent intervention strategies and raised need …

The availability of disease modifying therapies for spinal muscular atrophy (SMA) has
created an urgent need to identify clinically meaningful biomarkers. Biomarkers present a …

Background There is an unmet need for reliable biomarkers to predict disease severity,
prognosis, and treatment effect in patients with spinal muscular atrophy (SMA). The purpose …

Objectives Spinal Muscular Atrophy (SMA) presents challenges in (i) monitoring disease
activity and predicting progression,(ii) designing trials that allow rapid assessment of …

Background: Spinal muscular atrophy (SMA) is caused by bi-allelic, recessive mutations of
the survival motor neuron 1 (SMN1) gene and reduced expression levels of the survival …

Spinal muscular atrophy (SMA) is a lower motor neuron disease due to biallelic mutations in
the SMN1 gene on chromosome 5. It is characterized by progressive muscle weakness of …

Spinal muscular atrophy (SMA) is a monogenic disorder caused by loss of function
mutations in the survival motor neuron 1 gene, which results in a broad range of disease …

PURPOSE OF REVIEW This article provides an overview of the pathophysiology and clinical
presentations of spinal muscular atrophy (SMA) and reviews therapeutic developments …

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 …

Abstract Spinal Muscular Atrophy (SMA) is caused by autosomal recessive mutations in
SMN1 and results in the loss of motor neurons and progressive muscle weakness. The …