PTC Therapeutics announced that, based on recent feedback from the FDA, by mid-year they plan to re-submit an NDA for Translarna (ataluren) for Duchenne caused by nonsense mutations (also called premature stop codons). We will keep the community updated on the progress.
You can advance the care, treatment and cure for Duchenne muscular dystrophy. Contributions in any amount can truly make a difference and can be credited to a fundraiser or event from the list below.
We use cookies to ensure that we give you the best experience on our website. By continuing to use this site you are agreeing to accept our use of cookies. For more information about cookies please visit our Privacy Policy page.I Agree