Key Highlights
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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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