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.
Despite Favorable Safety, Olesoxime Falls Short Among Efficacy Markers for SMA
December 10th 2020Long-term follow-up of the treatment showed that while it has a favorable safety profile, the treatment does not offer significant efficacy for patients with spinal muscular atrophy (SMA) type 2 and non-ambulant type 3.
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Population Carrier Screening Increases Prenatal SMA Diagnoses, Reduces SMA Births
December 7th 2020Between the first introduction of population carrier screening in 2008 and its expansion to the full Israeli population in 2013, the mean rate of prenatal diagnoses per year was 4.66, and this rose to 7.75 between 2014 and 2017, a 66% increase.
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Case Report Shows Neuropathological, Molecular Findings of SMA Type 3 With Superficial Siderosis
November 28th 2020Researchers have published their postmortem report on a case of spinal muscular atrophy type 3 complicated by superficial siderosis, offering details on their neuropathological and molecular findings.
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Analysis Shows Utility of Tool to Detect Motor Function Change in SMA
October 17th 2020Analyzing data from a phase 2 study of patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA,) researchers found that the assessment is a valid, reliable, and responsive assessment of motor function ability for these patients.
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Survey Captures Baseline Experience of Patients With SMA
October 9th 2020A recent study about the impact of spinal muscular atrophy (SMA) on patients and caregivers may help to illustrate the impact of future improvements as new therapies take hold, but more targeted SMA quality-of life-outcome measures may be needed to fully the changes.
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Dr Mary Schroth Discusses At-Home Treatment Options for Patients With SMA
October 4th 2020Increasing at-home access to care and treatment options for spinal muscular atrophy (SMA) proved to be helpful during the coronavirus disease 2019 (COVID-19) pandemic, explained Mary Schroth, MD, FAAP, FCCP, chief medical officer at Cure SMA.
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New Research Offers Key Safety, Early Efficacy Data on Zolgensma in SMA
September 25th 2020A year after the FDA approved Zolgensma for the treatment of spinal muscular atrophy in children aged under 2 years without end-stage weakness, researchers shared safety and early efficacy data from the first 21 children treated with the gene therapy in Ohio.
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Improved Treatment Options for SMA Present New Opportunities, Challenges
September 5th 2020There are now multiple therapeutic options available for spinal muscular atrophy (SMA), a disease once considered incurable; however, there remains a need for additional treatments and better ways to predict treatment response.
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Socioeconomic Status Impacted Reproductive Carrier Screening Testing in Australia
September 1st 2020Reproductive carrier screening can identify conditions like spinal muscular atrophy, but this testing is often not done at all or not done before a woman gets pregnant, according to a new study.
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SMA Impacts Health-Related Quality of Life for Both Patients and Caregivers
August 28th 2020In addition to high health care and societal costs, spinal muscular atrophy (SMA) is associated with a deterioration in the health-related quality of life of both patients and their caregivers in 3 European countries.
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After FDA Approval, EMA Accepts Marketing Authorization Application for First Oral SMA Therapy
August 24th 2020The FDA approved the drug on August 7, and on August 17, the European Medicines Agency (EMA) accepted the marketing authorization application for the spinal muscular atrophy (SMA) treatment.
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Experts Discuss Symptoms, Types, and Treatments of SMA
September 22nd 2019A recent Peer Exchange series from The American Journal of Managed Care® brought together a panel of experts to discuss spinal muscular atrophy (SMA), including its clinical presentation, diagnosis of its various types, and the potential of new disease-modifying treatments. The panel was moderated by Peter L. Salgo, MD.
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FDA Approves Gene Therapy With $2.1M Price Tag for Spinal Muscular Atrophy in Pediatric Patients
May 24th 2019AveXis—a Novartis company—announced that it will work with payers to implement 5-year outcomes-based agreements and novel pay-over-time options. The company also said it will offer a patient program to support affordability and access.
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