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Multi-Center, Dose-Escalation Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta in OPMD Patients

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Trial Profile

Multi-Center, Dose-Escalation Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta in OPMD Patients

Status: Completed
Phase of Trial: Phase II

Latest Information Update: 03 Aug 2022

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At a glance

  • Drugs Trehalose (Primary)
  • Indications Oculopharyngeal muscular dystrophy
  • Focus Adverse reactions
  • Acronyms HOPEMD
  • Sponsors Bioblast Pharma

    • Most Recent Events

    • 26 Sep 2016 According to a Bioblast Pharma media release, results from this study including an extension study (Profile 700252783) are being presented at the annual scientific meeting of the Muscle Study Group Society (MSGS).
    • 25 May 2016 According to Bioblast Pharma media release, the company announced positive final results from the HOPEMD trial and presented final study results at Myology 2016 and the American Academy of Neurology (AAN) 2016 Annual Meeting.
    • 21 Apr 2016 Preliminary results of 24 weeks presented at the 68th Annual Meeting of the American Academy of Neurology.

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