Overview

• A 12-patient case series published in *The Lancet* highlights the safety and potential efficacy of ulefnersen in treating FUS-ALS, a rare and aggressive genetic form of ALS.
• One patient regained the ability to walk unaided and breathe without a ventilator, marking an unprecedented recovery in ALS treatment.
• A man treated pre-symptomatically has remained asymptomatic for three years, with improved muscle electrical activity.
• Patients experienced up to an 83% reduction in neurofilament light, a key biomarker of nerve damage, after six months of treatment.
• A global clinical trial is now in progress to validate these findings and potentially lead to regulatory approval of ulefnersen.