Björn Oskarrson

Stories

Custom RNA drug improves rare ALS in first personalized case

As of September 2026: Study co-author, reported results September 2026

A physician with a rare inherited form of amyotrophic lateral sclerosis (ALS) received a drug built around his own gene mutation, the first therapy designed for this genetic form of the disease. A year after his first dose, his neuron-damage biomarker fell into the normal range and he kept practicing medicine.

Updated Sep 21

Notable Quotes

Being able to develop a drug to target a specific person's mutation, test it in animals and then give it to the person within their lifespan was amazing.

from Custom RNA drug improves rare ALS in first personalized case · Updated Sep 21

We don't have decades of experience with it. We are hopeful that it will hold for their lifetime, but we do not know that.

from Custom RNA drug improves rare ALS in first personalized case · Updated Sep 21