Trusted Resources: Education
Scientific literature and patient education texts
Systematic Literature Review to Assess the Cost and Resource Use Associated With Spinal Muscular Atrophy Management
authors: Paracha N,Hudson P,Mitchell S,Sutherland CSsummary/abstract:
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 births. The traditional classification of SMA includes five types (Types 0-4 SMA) based on patient age at disease onset and the highest motor milestone achieved. Spinal muscular atrophy leads to progressive muscle denervation, skeletal muscle atrophy and loss of motor function and ambulation, though phenotypes vary along a disease continuum. Regardless of disease severity, or access to treatment, a multidisciplinary approach to care is required to ease the burden of disease. To date, limited global data exist regarding the cost and resource use associated with SMA management.organization: F. Hoffmann-La Roche Ltd, Grenzacherstrasse 124 Building 001/OG13, CH, 4070, Basel, Switzerland.
Spinal Muscular Atrophy With Respiratory Distress <br /> Type 1: A Novel Variant of IGHMBP2 GeneSpinal muscular atrophy (SMA) with respi...
Identification of Specific Gene Methylation Patterns During Motor Neuron Differentiation From Spinal Muscular Atroph...Spinal muscular atrophy is a progressive...
Neurofilament Levels in CSF and Serum in an Adult SMA Cohort Treated With NusinersenObjective: To retrospectively evaluate t...
A Novel CARM1-HuR Axis Involved in Muscle Differentiation and Plasticity Misregulated in Spinal Muscular AtrophySpinal muscular atrophy (SMA) is charact...
Spinal Muscular Atrophy: OverviewWhat is spinal muscular atrophy (SMA)?Sp...
Implications of Circulating Neurofilaments for Spinal Muscular Atrophy Treatment Early In Life: A Case SeriesThis longitudinal cohort study aimed to ...
Spinal Muscular Atrophy Type 2 (Intermediate SMA)Spinal muscular atrophy (SMA) is a genet...