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Duchenne Muscular Dystrophy clinical trials at UCSF

1 in progress, 0 open to eligible people

Showing trials for
  • Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)

    Sorry, accepting new patients by invitation only

    The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC sponsored clinical trial or treatment plan, and siblings of those participants (provided those participants have completed the placebo-controlled portion of the trial). The treatment will continue under this protocol until consent withdrawal by participants, withdrawal due to worsen condition after initiating ataluren treatment, withdrawal by investigator, withdrawal due to participant unable to tolerate ataluren, participant is eligible to participate in another ataluren nmDBMD clinical trial program initiated by sponsor, study is discontinued by the relevant regulatory authority and/or sponsor, or until ataluren becomes commercially available.

    San Francisco, California and other locations

Our lead scientists for Duchenne Muscular Dystrophy research studies include .

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