Amyotrophic lateral sclerosis (ALS) is a rare and life-threatening neurodegenerative disease characterized by progressive muscular paralysis reflecting degeneration of motor neurons. ALS is associated with substantial morbidity and severely impacts day-to-day functioning. The disease is relentlessly progressive.
There is still a high need for new treatments in ALS. In Europe, there has been no drug registered since riluzole 32 years ago.