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Censavudine is a small molecule nucleoside reverse transcriptase inhibitor (NRTI) originally developed for the treatment of HIV infection. It blocks the activity of HIV reverse transcriptase, thereby inhibiting viral replication. Censavudine is structurally related to stavudine but has been shown in preclinical studies to have improved efficacy and reduced toxicity compared to other NRTIs. The drug was initially developed at Yale University and later by Bristol Myers Squibb for HIV, but its development focus has shifted toward neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), Aicardi-Goutières Syndrome (AGS), progressive supranuclear palsy (PSP), frontotemporal dementia (FTD), and Alzheimer's disease. Its mechanism in these indications may involve modulation of immune responses, particularly reduction of interferon signaling in AGS. Censavudine is currently under clinical investigation by Transposon Therapeutics, with ongoing Phase II trials for several neurodegenerative conditions[1][2][4][5][8].
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