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.
Researchers Outline Management Strategies for Growing Adult SMA Population
March 12th 2022Due to advances in the treatment of spinal muscular atrophy (SMA), the lifespan of patients with the disease has increased significantly, allowing them to enter adulthood. However, the change in SMA prognosis has also led to a knowledge gap in the natural history, management, and treatment strategy for adult patients, according to the researchers.
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PEF May Be Preferred Measure of Respiratory Strength for SMA Follow-Up
March 4th 2022With clear differences at baseline and subsequent declines seen in a linear pattern, the researchers suggested peak expiratory flow may be a suitable outcome measure for longitudinal assessment of respiratory muscle strength in these patients.
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Researchers Determine What Patients, Caregivers Deem Meaningful Change in SMA Motor Function
February 12th 2022Through the use of patient testimonials as well as analysis of data from a study of patients with spinal muscular atrophy (SMA), researchers were able to describe patient-centered “meaningful outcomes” in the 32-item Motor Function Measure.
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Risdiplam May Be Superior for Certain SMA Type, According to Indirect Comparison
January 30th 2022Researchers compiled data from studies of oral Evrysdi (risdiplam), Spinraza (nusinersen), and Zolgensma (onasemnogene abeparvovec), finding evidence that risdiplam may be a favorable alternative to nusinersen for type 1 SMA.
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Multidisciplinary Care Emphasized for Pregnant Women With SMA
January 27th 2022According to researchers, this case of a patient with spinal muscular atrophy (SMA) from Indonesia was the first to use a collaborative, multidisciplinary approach that involved obstetric, neurology, pediatric, and anesthesiology departments at a hospital.
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Top 5 Most-Read Spinal Muscular Atrophy Stories of 2021
December 22nd 2021The top 5 most-read spinal muscular atrophy stories of 2021 on AJMC.com covered a variety of topics concerning the disease, including cost differences, the potential for combination therapies, and identifying biomarkers for treatment.
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New Imaging Approach May Assess Disease Burden in Patients With SMA
December 18th 2021If further studies confirm a benefit by using Multispectral Optoacoustic Tomography (MSOT), the technology can help forge a path toward a noninvasive, bedside, nonionizing approach to early visualization of disease as well as evaluation of disease burden and disease progression in patients.
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Scoliosis Surgery for Pediatric SMA1 Comes With Serious Complications, Study Says
December 11th 2021A case series of corrective surgeries in children with severe scoliosis due to spinal muscular atrophy (SMA) type 1 showed a positive success rate, but with significant postoperative complications.
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Pilot Program Underscores Importance of Newborn Screening to Identify, Treat SMA
December 2nd 2021Testing for spinal muscular atrophy in an Australian newborn screening program identified patients across socioeconomic and cultural demographics, mitigating inequity and providing patients with access to multidisciplinary treatment.
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Analysis of Nusinersen Shows Favorable Safety Profile in Adults With SMA
November 11th 2021Data on nusinersen’s safety profile in adult patients are more limited than in infant and adolescent patients, but a recent study showed positive results based on cerebrospinal fluid and blood sample parameters.
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