An interactive presentation focused on harnessing creativity in day-to-day life with inclusive activities, games and exercises, as well as utilizing adaptive technology/equipment to surpass expectations.
Paul, Debra and Hawken Miller discuss FUTURES 2022, current advancements in therapies and next generation investments as well as programs CureDuchenne offers to improve the quality of life for those impacted by Duchenne.
A brief introduction on the rationale behind mechanisms of endogenous dystrophin restoration, like exon skipping and read through agent therapies, followed by critical information and updates from the companies in this space. Moderated discussion included.
CureDuchenne Link is a centralized data hub which combines clinical data, biosamples, and patient reported data for individuals with Duchenne and Becker muscular dystrophy, as well as carriers. Information and samples provided by participants are coded and available for use by approved researchers across the world.