Myotonic dystrophy type 1 (DM1) is an inherited, progressive disease that mainly affects the muscles, heart, and nervous system. It occurs in about 1 in 8,000 people worldwide, with roughly 40,000 patients in the United States, 75,000 in Europe, and 15,000 in Japan. Mis-splicing of many important genes is said to be the underlying cause of DM1.
People with DM1 endure muscle stiffness (myotonia), muscle weakness, heart and breathing problems, fatigue, digestive issues, early cataracts, and difficulties with thinking or behaviour. In more severe cases, DM1 can shorten life expectancy, mainly because of heart and lung complications. There are no approved drugs for this rare genetic disorder.
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