PROCEEDINGS OF THE XVIII CONGRESS OF THE ITALIAN ASSOCIATION OF MYOLOGY

The broad clinical spectrum of myotonic dystrophy type 1 (DM1) creates particular challenges for both medical care and the design of clinical trials. Clinical onset spans a large continuum from birth to late adulthood, including highly variable symptoms regarding severity and nature of the affected organ systems. In the literature, this complex phenotype is divided from three grades (mild, classic, and severe) to four or five clinical categories (congenital, infantile/juvenile, adult-onset and late-onset forms), and no consensus is established so far