Platform Trials in ALS

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder characterized by progressive weakness of voluntary muscle. ALS is uncommon but not rare, with a lifetime risk of about 1 in 400. Nearly all patients with ALS will die from ALS, most from respiratory failure, and the median survival from onset of symptoms is 2 to 3 years. Treatment remains mostly supportive. Interventions stopping disease progression are nonexistent and those slowing progression unsatisfactory. There is a need to develop and quickly and cost-effectively evaluate better treatments.