Best Practices and Standard Protocols as a Tool to Enhance Translation for Neuromuscular Disorders

Recent years witnessed an exciting increase in the number of clinical trials for neuromuscular disorders, in particular for Duchenne Muscular Dystrophy and Spinal Muscle Atrophy. Given the high emotional impact of such developments for devastating diseases with an urgent medical need, it is particularly important to justify human trials on the basis of robust preclinical studies and to avoid a waste of hopes and of funds. This review focuses the discussion on the quality in the conduct clinically-oriented preclinical assessments in rare neuromuscular disease models and on the importance in reporting of preclinical confirmatory studies. Accordingly, it invites scientists, journal publishers and funding agencies to require quality standards to improve translatability of preclinical findings. Recent years witnessed an exciting increase in the number of clinical trials for neuromuscular disorders, in particularfor Duchenne Muscular Dystrophy and Spinal Muscle Atrophy. Given the high emotional impact of such developments fordevastating diseases with an urgent medical need, it is particularly important to justify human trials on the basis of robustpreclinical studies and to avoid a waste of hopes and of funds.This review focuses the discussion on the quality in the conduct clinically-oriented preclinical assessments in rare neuromusculardisease models and on the importance in reporting of preclinical confirmatory studies. Accordingly, it invites scientists,journal publishers and funding agencies to require quality standards to improve translatability of preclinical findings.