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↗️ The NEXT Report - Become a Citizen Scientist

  • 1.  ↗️ The NEXT Report - Become a Citizen Scientist

    Posted 03-12-2023 17:51

    Here's another one of the great stories from The NEXT Report by Global Genes. #researchcollabortations #researchReadiness #patientvoice  

    Read the full report here. 

    The efforts of Chion Foundation’s President and Founder Lara Pullen and TREND Community co-founder and CEO Maria Picone, parents of children with Prader-Willi syndrome, have changed the understanding of that disease. #PraderWilli #citizenScientist 

    Prader-Willi is a complex genetic condition characterized by obesity, excessive hunger, low muscle tone, and developmental and intellectual disability. The work Pullen and Picone did highlighted the sleep apnea and daytime sleepiness that is associated with the condition and identified pitolisant, a recently approved drug, that had the potential to benefit these patients. Harmony Biosciences, the company that pro- duces the drug, is conducting a phase 2 clinical study of the drug in people with Prader-Willi as a result of their efforts. #drugrepurposing 

    Daniel DeFabio

    Global Genes
    Director of community engagement