Tagged therapy

Exon Skipping Therapy

Duchenne muscular dystrophy (DMD) is a lethal genetic disorder that shortens patients’ lifespans and results in progressive skeletal and cardiac muscle degeneration. The symptoms emerge at the onset of muscle growth in boys 2-5 years old. They grow up bound to wheelchairs and ventilators, and eventually succumb to an early death after cardiac failure (2)….

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