Ulefnersen for treating amyotrophic lateral sclerosis

Ulefnersen is currently being developed for the treatment of patients with amyotrophic lateral sclerosis (ALS) who have alterations in the fused in sarcoma (FUS) gene. ALS is a neurological disorder that affects motor neurons. Motor neurons are the nerve cells in the brain and spinal cord that control voluntary muscle movement and breathing. ALS is a form of motor neurone disease in which the nerves that control movement gradually stop working, leading to muscle weakness, stiffness, and wasting, affecting the ability to walk, speak, eat, and breathe. Symptoms worsen over time, and the condition is life-shortening. FUS-related ALS is a rare but more aggressive form of the disease that often occurs earlier in life and progresses quickly. There are currently no specific treatments available for people with FUS-related ALS, highlighting a significant unmet clinical need.