Trusted Resources: Education
Scientific literature and patient education texts
Spinal Muscular Atrophy: Types and Genetics
source: Novartis Gene Therapies, Inc
Spinal Muscular Atrophy (SMA)
SMA is a rare and devastating genetic disease caused by a lack of a functional survival motor neuron 1 (SMN1) gene, resulting in the rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement.
SMA is a rare condition, yet it is the leading genetic cause of infant death. It is imperative to diagnose SMA and begin treatment, including proactive supportive care, as early as possible to halt irreversible motor neuron loss and disease progression.
Let’s Start From the BeginningScarlet was born at 40 weeks and 1 day. ...
Spinal Muscular Atrophy Patient iPSC-Derived Motor Neurons Display Altered Proteomes at Early Stages of Differentiat...Spinal muscular atrophy (SMA) is an auto...
Nusinersen for Spinal Muscular Atrophy in the United States: Findings From a Retrospective Claims Database AnalysisSpinal muscular atrophy (SMA) is a rare,...
Managing Pregnancy in a Spinal Muscular Atrophy Type III Patient in Indonesia: A Case ReportSpinal muscular atrophy (SMA) is a genet...
How One Mother Is Raising Awareness for Spinal Muscular Atrophy – A Leading Genetic Cause of Infant DeathWhen Nicole Clark’s son Brady was born...
FDA-Approved Treatment Provides Life-Saving Miracle for Girl With Spinal Muscular AtrophyAlex says her daughter made it to Norton...
Despite Taking Spinraza, SMA Type 1 Children Show Nonmotor ProblemsDespite its well-reported benefits in mo...