Antisense Oligonucleotides (ASO) Treatment

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Antisense Oligonucleotides (ASO) Treatment

This treatment would reduce the expected toxicity to cells from the mutated tubulin by using an approach that captures the mutated RNA before it becomes a damaged protein. Unlike the AAV treatment, ASO treatment is not a permanent cure and requires ongoing distribution of the serum to the patient to maintain the reduction of the toxicity. Therefore, while we wait for a permanent cure, the ASO will extend the lives of affected patients. Read more about ASOs in Nature: here.

Synaptix Bio is the leading biotech advancing the preclinical trial process with the FDA and EMA (British equivalent).

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