• Report - 1st day of the International Congress on the Effects of COVID-19 in People with Rare Diseases

    The opening of the congress was led by Patricia García, medical director of the Garrahan Hospital, a reference centre for rare diseases in Argentina. García explained the challenge that COVID-19
  • An Interview with Marjolein van Kessel — President of Naevus Global

    Thank you, Marjolein, for taking the time to be interviewed! Could you please give us a bit of background about congenital melanocytic naevi and the issues associated with them? A congenital
  • International Congress on the Effects of COVID-19 on People with Rare Diseases

    The International Congress on the Effects of COVID-19 on People with Rare Diseases will be held on November 9-11th 2020 and it is organized by the Sant Joan de Déu Barcelona Children's Hospital
  • World Duchenne Awareness Day: "Together, we are stronger"

    This year's awareness campaign ‘Together, we are stronger’ was translated in 15 languages and collected over 1,000,000 views globally. This impressive number was reached due to an amazing
  • Data for a difference — The Share4Rare research feature for patient organisations

    Violeta Astratinei is a Ph.D. biologist with a background in environmental research. After she lost her sister to melanoma in 2014, she became a core member of Melanoma Patient Network Europe, MPNE
  • Gemma Marfany: "Researchers, patients and families must come together to fight rare diseases"

    Gemma Marfany is a professor in the Department of Genetics at the University of Barcelona (UB) and head of Unit U718 at CIBERER, the Spanish biomedical research centre for rare diseases. She is also a
  • World Duchenne Awareness Day 2020: Duchenne and the brain

    Learning and behavioural challenges in DMD and BMD Both conditions are characterised by progressive muscle breakdown. A certain part of the DNA is missing, duplicated, or changed so the code cannot be
  • New webinar — Patient advocates fostering research in oncology: the Share4Rare experience

    One of the pillars of Share4Rare is to enhance collaborative research. For that purpose, at the end of 2019 the platform started piloting four research projects concerning several groups of rare
  • ASCO 2020 — Free learning opportunity for patients!

    ASCO Annual Meetings are great opportunity for healthcare professionals and patients advocates all over the world to follow the latest scientific news in cancer research. Exceptionally this year, in
  • New registry to better understand how COVID-19 affects people with rare diseases

    In the current global pandemic due to COVID-19, patients affected by a rare disease are a doubly vulnerable group: on the one hand, many of the rare pathologies put patients at a higher risk of
  • What is a patient registry?

    A patient registry collects information about patients who are affected by a particular condition. Registries are databases containing quantitative and qualitative data about the patients. In rare
  • Virtual coffee with patient organisations and Share4Rare: 'Advancing a Patient-Centric Research Agenda'

    Research is at the heart of solutions for all patients. However, it is not always conducted with the patient in mind. Patient organisations are increasingly invited to submit evidence to healthcare