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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Motor Function Continues to Improve in Second Year of Risdiplam Treatment for SMA
November 26th 2022Detailing findings from the secondary end points of the FIREFISH trial at 24 months, the researchers found that risdiplam treatment was associated with continued improvements in motor function and the achievement of development motor milestones.
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Certain CSF Biomarker May Foretell Nusinersen Response in Children With SMA
November 22nd 2022Using data from patients with spinal muscular atrophy (SMA) type 1 and 2, researchers observed that chitotriosidase 1 levels in cerebral spinal fluid (CSF) changed over time after treatment with nusinersen, suggesting that the change could be indicative of treatment response.
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Balancing Hopes for a Cure for SMA, DMD With Realistic Expectations
September 21st 2022A new study looks at the balancing act physicians and families must carry out when managing expectations at a time of great scientific advancement in spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD).
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Patients and Parents Impacted by SMA May Be Optimistic About Prenatal Testing, Therapies
September 2nd 2022As the feasibility of phase 1 clinical trials for prenatal spinal muscular atrophy (SMA) therapies is explored, patient and parent input on prenatal testing and possibly treatment is a valuable tool for guiding research discussions.
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Nusinersen Linked With Modest Improvements in Adult SMA
August 26th 2022The patients, from 5 referral centers, were followed for at least 6 months and showed modest improvements in both motor and functional scales when treated with the antisense oligonucleotide for spinal muscular atrophy (SMA).
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