Tag: Antisense Oligonucleotide
-
Breakthrough Antisense Oligonucleotide Therapy Offers Hope for Ultra-Rare Diseases: Personalized Medication Transforms Lives in Pioneering Medical Approach
In 2016, Luke Rosen and Sally Jackson faced a dire situation when their daughter Susannah was diagnosed with an ultra-rare genetic condition, a mutation in the KIF1A gene. Crooke, a pioneer in antisense oligonucleotide (ASO) technology, promised a personalized medication for Susannah Traditional medical paths offered no hope, predicting a downward spiral toward death within…