A groundbreaking experimental therapy for a rare, aggressive form of ALS caused by FUS gene mutations has produced surprising clinical improvements in some patients, including restored mobility and extended survival. The treatment uses antisense technology to silence the FUS gene, reducing toxic protein buildup in motor neurons.
Two patients in a recent case series showed remarkable responses—one regained the ability to walk and breathe unassisted, while another has remained symptom-free for three years. The therapy also significantly reduced neurofilament light levels, a biomarker of nerve damage.