Antisense Oligonucleotide Therapy for an Ultra-Rare Neurological Variant: A Case Report
DOI:
https://doi.org/10.5281/zenodo.22648851Keywords:
Antisense Oligonucleotide Therapy for an Ultra-Rare Neurological Variant, Translational medicine; Evidence synthesis;, Clinical implementation; Health services researchAbstract
Antisense Oligonucleotide Therapy for an Ultra-Rare Neurological Variant is an area of active development in translational
medicine, with evidence that has accumulated unevenly across its component questions. We review the current evidence base for
antisense oligonucleotide therapy for an ultra-rare neurological variant, spanning analytical, clinical, implementation,
governance, and economic dimensions, focusing on where practice has converged and where it continues to vary, and set out the
practical considerations relevant to a service considering its adoption. We conclude that selective, context-specific use is best
supported by the evidence currently available, rather than uniform adoption or avoidance
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