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Clinical trials

  • Pfizer Early Stage GT

    The study will evaluate the safety and dystrophin expression following gene therapy in boys with Duchenne Muscular Dystrophy (DMD). It is a single-arm, non-randomized, open-label study

    Investigator
    Barry J Byrne
    Ages
    2 Years - 3 Years
    Sexes
    Male
  • Edgewise-210

    The LYNX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics and biomarkers in children with Duchenne muscular dystrophy including a randomized, double-blind, placebo-controlled part A, followed by an open-label part B.

    Investigator
    Renata Shih
    Ages
    4 Years - 9 Years
    Sexes
    Male
  • Givinostat Long Term Study

    This is an open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD (Duchenne's muscular dystrophy) patients who have been previously treated in one of the GIVINOSTAT studies.

    Investigator
    Barry J Byrne
    Ages
    7 Years - N/A
    Sexes
    Male