Fleur Garton

New Capabilities

As of September 2026: External expert commenting on implications for broader ASO treatment

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