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A new study found that combining two treatments—one that fixes the faulty SMN2 gene and another that blocks an enzyme called HDAC6—greatly improved muscle strength and survival in a mouse model of spinal muscular atrophy (SMA). This is important because it offers a potential way to help SMA patients who still face muscle weakness even after receiving newer gene therapies.

This week’s Medicine Key Highlights

This week’s Medicine Key Highlights

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This week’s Medicine Key Highlights

Last updated: May 4, 2026 10:17 am
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Combining SMN2 splicing modifiers with a drug that inhibits HDAC6 strongly improved muscle strength, mass, and longevity in a mouse model of spinal muscular atrophy (SMA). This combination approach could help treat persistent muscle weakness in SMA patients who currently receive gene therapy.
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A new review highlights the strong, bidirectional link between NAD+ metabolism and the circadian rhythm system in dementia, including Alzheimer’s disease. This connection influences sleep and cognitive symptoms, suggesting that strategies targeting NAD+, such as specific precursors or timed light exposure, could offer new therapeutic avenues.
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Appropriate use recommendations for the Alzheimer’s drug lecanemab have been published in the UK over a year after its regulatory approval, highlighting practical and organizational challenges. The commentary notes the significant hurdle of securing reimbursement from the National Institute for Health and Care Excellence (NICE) for this new class of amyloid-targeting therapy.
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Next Article A new study found that combining two treatments—one that fixes the faulty SMN2 gene and another that blocks an enzyme called HDAC6—greatly improved muscle strength and survival in a mouse model of spinal muscular atrophy (SMA). This is important because it offers a potential way to help SMA patients who still face muscle weakness even after receiving newer gene therapies.
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