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Latest news

  • Sarepta announce early, preliminary gene therapy trial results

    Sarepta therapeutics has announced preliminary results of a gene therapy trial that is using a harmless virus to deliver a small but functional dystrophin gene (micro-dystrophin) to the leg muscles of people living with Duchenne. The early results are encouraging, but it is too early to say whether the potential treatment will prove to be safe or effective. Read more

  • CEO attends EURORDIS Summer School

    Last week our incoming CEO, Shelley Simmonds, completed a 5 day intensive training programme in Barcelona, having been selected to take part in the 'Expert Patient and Researcher EURORDIS Summer School'. Read more

  • Solid Biosciences announce FDA have removed clinical hold from their gene therapy trial

    Solid Biosciences Announces FDA Removes Clinical Hold on SGT-001 - Activities to Resume Enrollment in IGNITE DMD Phase I/II Clinical Trial are Underway Read more

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