Friedrich Baur Stiftung

Could NQO1 genotypes impact the results of clinical trials with idebenone?

Carelli et al. argue that new insights into how NQO1 influences the response to idebenone warrant reevaluating the results of previous clinical trials, including those in Friedreich ataxia and Duchenne muscular dystrophy, using patient stratification by NQO1 genotype.

Psychological symptoms in individuals with Spinal Muscular Atrophy (SMA) and their caregivers – results from a nation-wide study in Germany

Abstract Background This study investigates the prevalence and associations of psychological symptoms in individuals with Spinal Muscular Atrophy (SMA) and their caregivers, utilizing data from a cross-sectional, observational assessment conducted in Germany. Participants were re...