A conditional marketing authorization has been recommended for Translarna (ataluren) , an orphan-designated medicine, by the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP), for the treatment for Duchenne muscular dystrophy caused by nonsense mutations. An orphan drug is a pharma agent developed specifically to treat a rare medical condition that is also known as orphan disease. Translarna is to be used in patients aged five years and older ...
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