PTC Therapeutics announced that Translarna – ataluren – has been granted marketing approval from the Brazilian National Health Surveillance Agency under rare diseases procedure, for the treatment of ambulatory children five years and older with Duchenne muscular dystrophy caused by a nonsense mutation. Patients now can have access to a treatment that targets the underlying cause. Duchenne muscular dystrophy is a rare, irreversible, and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-twenties due to heart and respiratory failure.
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