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
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