Of rAAV and Men: From Genetic Neuromuscular Disorder Efficacy and Toxicity Preclinical Studies to Clinical Trials and Back

Abstract excerpt

Neuromuscular disorders are a large group of rare pathologies characterised by skeletal muscle atrophy and weakness, with the common involvement of respiratory and/or cardiac muscles. These diseases lead to life-long motor deficiencies and specific organ failures, and are, in their worst-case scenarios, life threatening. Amongst other causes, they can be genetically inherited through mutations in more than 500...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Muscle Physiology and Disorders
• Share to Neurogenetic and Muscular Disorders Research
• Share to Virus-based gene therapy research