• VISION-DMD App for boys with Duchenne Muscular Dystrophy

    The Vision-DMD project aims to advance and accelerate the clinical development of the orphan drug Vamorolone —also known as VBP15— for the treatment of Duchenne Muscular Dystrophy (DMD) in ambulant
  • How to build an infrastructure for translational research

    On 11th December 2018, Rebecca Leary, EURO-NMD Project Manager and member of the TREAT-NMD Secretariat and Cathy Turner, DMD Liaison and TACT Coordinator at the John Walton Muscular Dystrophy Research
  • Share4Rare Webinar IV: “Creating the infrastructure for translational research”

    On Tuesday December 11, 7pm CET, Share4Rare hosts a webinar that will describe the objectives, goals and achievements of the specialist network TREAT-NMD. Established in the neuromuscular field TREAT
  • Share4Rare had its place in the Informative Session for Patients and Families with Neuromuscular Diseases at Hospital Sant Joan de Déu Barcelona

    Last Saturday, October 27th, the Share4Rare project was presented at the 5th Informative Session for Patients and Families with Neuromuscular Diseases that is annually celebrated at Hospital Sant Joan
  • How to go from a good idea to a great one: a case study in co-creation

    The digital environment is shaping the way we interact and interpret the healthcare environment. It has the power to erase borders, overcome old challenges and bridge the gap between very different
  • Poster prize for Share4Rare!

    Share4Rare received an "Innovative project" prize on their poster the at the Better Medicines for Children Conference in Brussels! This conference is organized by the European Forum for Good Clinical
  • Share4Rare Webinar III: “Fundraising for Nonprofits”

    Nonprofit organizations are likely to have limited time and resources to accomplish maximum return on investment. Effective fundraising has the potential to increase donations, grants and gifts. This
  • 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
  • How to guarantee quality in translational research for neuromuscular diseases

    It is not easy to go from basic drug research to clinical trials on humans, and for rare neuromuscular diseases the scenario is even harder. Besides, not always all the projects arrive to the human
  • Share4Rare Webinar II: “Following Scientific News for Patient Advocates”

    Patient advocates are in contact with many patients and increasingly involved stakeholders in the health debate. Understanding the latest scientific findings in their respective fields helps advocates
  • Share4Rare Webinar: "Social media in Rare Diseases"

    Social media is a cost-effective way for patient organizations and nonprofits to reach your audience and tell your story. In fact, over four in ten of the world’s population use social media platforms