Researchers Identify Potential Biomarkers for SMA Motor Improvement Following Nusinersen Treatment
November 17th 2023A retrospective, exploratory analysis identifies biomarkers predictive of spinal muscular atrophy (SMA) response to nusinersen and suggests the efficacy of using machine learning algorithms to anticipate patient outcomes.
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Nusinersen Modulates Neuroinflammation in Severe SMA, Study Says
March 4th 2023Researchers of the multicenter study looked at the effect of nusinersen among 48 patients with spinal muscular atrophy (SMA) from 2 Italian hospitals, finding more active immune systems among the patients with SMA1, the most severe form of the disorder.
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NGS-Based Assay Shows Feasibility, Potential Cost Savings in Newborn SMA Screening
February 18th 2023Investigators note that although the overall cost for implementing next-generation sequencing (NGS) testing would be more than implementing current polymerase chain reaction (PCR)–based testing, the assay may be more cost effective overall because it screens for multiple conditions.
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Real-World Data Show Most Nusinersen Doses Administered on Time
February 17th 2023The data showed that most doses of nusinersen were received on time while also underscoring the importance of being meticulous with the methodological approaches used with real-world databases for evaluating treatment patterns.
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Improving SMA HRQOL Must Address Patient and Caregiver Well-being
February 7th 2023Data from 2 prospective studies showed correlations between impaired patient health-related quality of life (HRQOL) and disease severity, as well as correlations between the health of caregivers and their HRQOL and caregiver burden.
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Screening for SMA in Newborns Alleviates Functional Burden, Associated Comorbidities
January 17th 2023A study found that screening for spinal muscular atrophy (SMA) in newborns paired with early access to disease-modifying therapies was effective in alleviating the burden and comorbidities of the disease.
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Symptoms That Contribute to SMA Disease Burden Clarified in New Study
January 12th 2023Qualitative interviews conducted with caregivers of children with spinal muscular atrophy (SMA) found that the symptoms with the greatest effect on disease burden were not always the most prevalent symptoms in patients.
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Improved Implementation Strategies Can Further Advance SMA Care, Researchers Say
January 7th 2023The diagnosis and treatment of a rare disease like spinal muscular atrophy (SMA) would benefit greatly from using implementation science to reduce the variation that exists in screening and therapy, according to researchers.
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Patients With SMA, Family Members Need Empathetic Attitude From Diagnosing Physician
January 6th 2023Patients who receive a diagnosis of spinal muscular atrophy (SMA) require an empathetic approach from their primary care physician when receiving the diagnosis and evaluating options, according to recent study findings.
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Severity of SMA Muscle Impairment Prior to Treatment May Have Implications for Nusinersen Efficacy
December 26th 2022The findings, which the researchers say highlight the importance of early diagnosis and initiation of treatment, come amid efforts to determine efficacy trends among the several treatment options for the condition.
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Repeated SMA Treatment With Nusinersen Does Not Result in Consistent Increase in Immune Responses
December 23rd 2022The findings, say the researchers, confirm that repeated intrathecal injections of the gene-targeting therapy are safe, adding to the limited data on the long-term effect and safety of the treatment approach in neurological diseases.
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