Clinical investigation of medicinal products for the treatment of amyotrophic lateral sclerosis - Scientific guideline

This document provides guidance on the clinical investigation of medicinal products for the treatment of myotrophic lateral sclerosis. It focuses on the design of studies for disease-modifying as well as symptomatic treatments, the choice of meaningful outcome parameters and the clinical relevance of functional tests of disability including motor and respiratory functions and their relationship to survival.

Keywords: Amyotrophic lateral sclerosis (ALS), motor neuron disease