December 31st 2023
This qualitative study conducted interviews with caregivers of patients with spinal muscular atrophy (SMA) to explore their unique experiences and challenges in navigating health systems to access disease-modifying therapies.
Nonadherence to Nusinersen Treatment Increases Health Care Utilization, Costs in SMA
November 5th 2021Results of a retrospective claims database analysis suggest that adherence to scheduled intrathecal nusinersen injections may lead to improvements in comorbidities, health care utilization, and costs for spinal muscular atrophy (SMA) types 1, 2, and 3.
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Study Evaluates Potential Cerebrospinal Fluid Biomarkers in SMA Treatment
October 30th 2021The single-center study explored cerebrospinal fluid neurofilament and protein levels in patients with spinal muscular atrophy (SMA) undergoing treatment with nusinersen in an effort to identify useful biomarkers as treatment options expand.
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More SMA Biomarkers Are Needed to Gauge Disease Progression, Therapy Response
October 21st 2021A review of current molecular and electrophysiological biomarkers in spinal muscular atrophy (SMA) concluded that more exploration is necessary to find noninvasive, yet accurate, measures of disease progression and therapy response.
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Subcutaneous Intrathecal Catheter Allows for Effective Administration of Nusinersen in SMA
October 14th 2021A novel indwelling subcutaneous intrathecal catheter circumvented the complex spine anatomy of patients with spinal muscular atrophy (SMA), a challenge when giving nusinersen via intrathecal injection.
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Modern Gene Therapies Show Promise in Motor Neuron Disorders
October 3rd 2021A review of current research and drug development in motor neuron disorders such as amyotrophic lateral sclerosis and spinal muscular atrophy shows progress in the identification of treatment pathways and therapies.
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Study Clarifies Prevalence and Incidence of SMA in Japan
September 16th 2021An epidemiological investigation of spinal muscular atrophy (SMA) in Japan provided clarity on the genetic condition’s prevalence and incidence in the country and highlighted the importance of newborn screening for SMA
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Improving Outcomes-Based Managed Entry Agreements for SMA Therapy
September 12th 2021Innovative therapies for rare diseases such as spinal muscular atrophy (SMA), when paid for by public budgets in the European Union and elsewhere, are often managed by agreements between payers and drug companies, but details can be hard to discern.
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Researchers Highlight Emerging Concepts Underlying Selective Neuromuscular Dysfunction in SMA
March 27th 2021Conducting a review of data published over a 25-year period, researchers highlight the importance of this type of research they say offers insight that drives the success of SMA treatment.
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Poster Presentations Demonstrate New Safety and Efficacy Data on Zolgensma for SMA
March 19th 2021Spinal muscular atrophy treatment Zolgensma was found to be safe and effective for long-term use and use in presymptomatic patients, according to posters presented at the 2021 MDA Virtual Clinical & Scientific Conference.
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Study Offers Snapshot of Successes and Challenges of Teens and Young Adults With SMA
March 14th 2021Results of a survey depicting the ways that spinal muscular atrophy (SMA) affects the lives of adolescents and young adults revealed areas of support that need to be addressed to ensure that patients are able to live their best possible lives.
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