Congenital Muscular Dystrophy (CMD)
CMD refers to a group of inherited neuromuscular disorders which frequently affects infants or young children. CMD is a devastating condition characterized by progressive loss of muscle tissue which in severe forms can already affect newborn babies (“floppy infant syndrome”). Severe forms of CMD cause immobility frequently at young age and reduced life expectancy. Important aspects of disease management include orthopedic surgery of scoliosis as well as supplementary nutrition to avoid malnutrition and ventilatory support.
There is currently no effective pharmaceutical treatment available or in advanced clinical development for CMD. In the absence of recent epidemiological studies the British Muscular Dystrophy Campaign estimates that one in every 20,000 – 50,000 children is born with CMD.