Advancements in RNA Medicine for Muscle Diseases

This chapter discusses the latest developments in treatments for myotonic dystrophy, focusing on a promising drug in phase three clinical trials. It highlights significant improvements in muscle strength and quality of life for patients, along with the challenges and responsibilities involved in drug development. Furthermore, it addresses ongoing research in Facioscapulohumeral Muscular Dystrophy (FSHD), emphasizing community engagement and corporate responsibility in the biotech sector.