Method for treating nonsense mutation mediated duchenne muscular dystrophy in pediatric patients
AssigneePTC Therapeutics, Inc.
InventorsRonald KONG, Joseph William McIntosh
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Abstract
Provided herein is a method for ameliorating or managing nonsense mutation mediated Duchenne muscular dystrophy (nmDMD) in a pediatric patient in need thereof comprising, administering an effective amount of ataluren to the patient.
Claims8
A method for ameliorating or managing nonsense mutation mediated Duchenne muscular dystrophy (nmDMD) in a human pediatric patient having nmDMD comprising, orally administering to a patient having an age in a range between greater than or equal to two years of age and an age less than five years of age a therapeutically effective amount of ataluren based on patient weight to attain an average plasma concentration in a range of at least about 1 μg/mL to about 20 μg/mL during a 24 hour time period.
Dependencies are inferred from the claim text.