Man with rare motor neuron disease improves on first RNA drug aimed at his mutation
A man with an uncommon type of motor neuron disease saw his symptoms improve and kept working as a physician a year after becoming the first person to receive a drug designed to target his mutation. The antisense oligonucleotide therapy goes after RNA from the mutated gene and cuts how much protein is made. Results appear in Med this week. Steve Vucic, a neurologist at the University of Sydney in Australia, called it an "exciting first step" and said it needs testing in more people.
Roughly 5 to 10% of people with ALS have a known genetic mutation. Fleur Garton of the University of Queensland says similar antisense therapies are the future of treatment for many people with the disease.
Written by the Genomes desk from the primary source cited and linked above and checked against it. Research use only; not medical advice. Corrections: [email protected].