• WDO Member Meeting

    The main aim of the WDO Member Meeting was to gather patient organizations, scientists, researchers and experts working in the Duchenne field to share and build on each other’s knowledge to create
  • RareHacks: uniting experts to build the future of the rare disease community

    What is RareHacks? RareHacks is a hackathon event that brings you the opportunity to apply data science tools , including machine learning and natural language processing algorithms, to a real
  • The POWER-tool: a new effective method that helps involving patients in clinical trial design

    Research on rare diseases is often difficult to perform mainly because there are only a small number of cases to study, resulting in a vaguely representative statistical analysis of the data. For
  • Cancer therapies assessment indicators need to be reviewed according to a novel study

    A recent publication on JAMA Internal Medicine refutes any association between progression-free survival (PFS) and health-related quality of life ( HRQoL) in cancer patients. In other words, delaying