Background Spinal muscular atrophy (SMA) is a rare and devastating condition for which
new disease-modifying treatments have recently been approved. Given the increasing …

Spinal muscular atrophy (SMA) is the most common genetic disease affecting infants and
young adults. Due to mutation/deletion of the survival motor neuron (SMN) gene, SMA is …

Objective The aim of the study was to report 12‐month changes after treatment with
nusinersen in a cohort of 85 type I spinal muscular atrophy patients of ages ranging from 2 …

Aim To compare the societal financial costs and quality of life (QoL) of untreated patients
with spinal muscular atrophy (SMA) and treated patients identified because they presented …

Aims: To estimate healthcare resource utilization (HRU) and costs among patients with
spinal muscular atrophy (SMA) type 1 (SMA1) in real-world practice, overall and among …

Objectives The objective of our study was to conduct a systematic literature review of
estimates of costs of illness of spinal muscular atrophy (SMA). Methods We searched …

We report the development of a new disease registry on SMA as the result of a collaboration
among three national networks in United States, Italy, and United Kingdom in partnership …

Background Spinal muscular atrophy (SMA) is a severe neuromuscular disease that is
inherited in an autosomal recessive manner with an estimated incidence of 1 in 10,000 live …

Background: Spinal muscular atrophy (SMA) is a rare genetic neuromuscular disease.
Objective: Characterize direct costs associated with SMA management. Data source: Truven …

Introduction The recent advent of disease-modifying therapies (DMTs) has dramatically
changed the treatment landscape of spinal muscular atrophy (SMA), and the multifaceted …