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Top 5 Most-Read Spinal Muscular Atrophy Content 2023
This year’s most-read spinal muscular atrophy(SMA) articles explored mental health, new technologies, caregiver well-being, and more.
The top 5 most-read spinal muscular atrophy (SMA) articles on AJMC.com this year included insights into caregivers’ experience, the development of novel genetic screening methods, the most effective tools for educational videos on the disease, and the mental well-being of young children with SMA.
Here are the top 5 SMA pieces of 2023.
5. Improving SMA HRQOL Must Address Patient and Caregiver Well-being
The impacts of an SMA diagnosis and the subsequent care extend beyond the patient. In a prospective study utilizing patient-reported outcome measures, researchers found the well-being of both a patient with SMA and their caregivers are closely associated with one another.
4. NGS-Based Assay Shows Feasibility, Potential Cost Savings in Newborn SMA Screening
Investigators have explored potential cost-saving avenues in the realm of genetic testing in SMA. Currently, polymerase chain reaction testing is a standard screening method in SMA; however, researchers found that utilizing next-generation sequencing (NGS) testing could be more cost-effective in the long run while additionally screening for various conditions.
3. Researchers Highlight Utility of Long-Read Method for SMN1, SMN2 Profiling in SMA
This year, researchers emphasized the value of an informatics method in identifying carriers of the SMN1 and SMN2 genes, as well as detecting pathogenic variants. This approach was developed using PacBio HiFi data and yielded effective results in screening for SMA-related gene haplotypes.
2. YouTube Videos Have Mixed Quality, Reliability for Educating Patients About SMA
Researchers conducted an analysis on the factors contributing to better educational outcomes in YouTube videos on SMA. Across the spectrum of video categories, results indicated that infographics had a higher influence on viewer learning.
1. Children With SMA Have Increased Anxiety, Depression
In this cross-sectional study, researchers investigated the behavior patterns in young children diagnosed with SMA compared with children enduring other chronic non-neurological diseases. Overall, children with SMA exhibited higher incidences of depression and anxiety; however, these results varied depending on the type of SMA diagnosis.
LLMs Show Promise, But Challenges Remain in Improving Inefficient Clinical Trial Screening
July 31st 2025Large language models (LLMs) such as GPT-3.5 and GPT-4 may offer a solution to the costly and inefficient process of manual clinical trial screening, which is often hindered by the inability of structured electronic health record data to capture all necessary criteria.
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Trump Directs Pharma Companies on Cutting Drug Prices Under Most-Favored-Nation Order
July 31st 2025President Donald Trump has sent letters to pharmaceutical companies, aiming to compel them to lower drug prices in the US to match the lowest prices offered in other developed nations, a move that could significantly reduce costs and disrupt the current system of pharmacy benefit managers.
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Laundromats as a New Frontier in Community Health, Medicaid Outreach
May 29th 2025Lindsey Leininger, PhD, and Allister Chang, MPA, highlight the potential of laundromats as accessible, community-based settings to support Medicaid outreach, foster trust, and connect families with essential health and social services.
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Trends in Insulin Out-of-Pocket Costs and Use Disparities, 2008-2021
July 31st 2025Given trends in cost and use, insulin out-of-pocket cost reduction policies would be more efficient if they targeted members in high-deductible health plans with savings options and low-income patients.
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Linvoseltamab Added as Preferred Agent in Newest MM Practice Guidelines
July 31st 2025On July 2, linvoseltamab-gcpt (Lynozyfic; Regeneron) received an accelerated approval from the FDA in relapsed/refractory multiple myeloma (MM), and the most recent update to the National Comprehensive Cancer Network guidelines for MM has added the BCMA-targeted bispecific antibody as a preferred treatment option.
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