ASO TREATMENT

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.

Principal Investigator

A team of researchers at Children’s Hospital of Philadelphia (CHOP) led by Dr. Adeline Vanderver is now at the clinical trial readiness stage of their research for a cure using ASOs.

Bio Tech

Synaptix Bio is leading biotech advancing the preclinical trial process with the FDA and EMA (British equivalent). Click on the link below to learn more about the trial.