• #S4RMonth activity: How to navigate psychological care in neuromuscular conditions

    The field of rare neuromuscular diseases houses many types of conditions, each with its own disease expression. One thing they all have in common is that being or caring for an individual with a
  • Meeting the partners — World Duchenne Organization

    The World Duchenne Organization (WDO) is one of the two patient organisations joining Share4Rare as project partner. Dimitrios Athanasiou, WDO board member and father of a boy with Duchenne, did not
  • An interview with Jeffrey Galuidi — Father of a young boy with congenital myasthenic syndrome

    Jeffrey Galuidi is Jackson’s father, a young boy with congenital myasthenic syndrome (CMS), a rare neuromuscular disease. “Jackson was diagnosed with a general muscle wasting condition a couple of
  • The Share4Rare research project on neuromuscular rare diseases: analysing how the disease affects employment and education

    The Share4Rare platform is growing in strength, with over 900 users validated since April 2019. This growing community of rare disease patients and families is providing a secure environment for
  • Duchenne Patient Academy 2020: a leading worldwide patient advocacy event on neuromuscular conditions

    Duchenne Patient Academy works in partnership with leading DMD patient organisations to set a strong patient advocacy base for patient organisations and the Duchenne and Becker community at large. We
  • The MRC Centre for Rare and Neuromuscular Diseases Biobank

    The principal objective of the MRC Biobank is to collect biomaterial from patients affected by neuromuscular disorders with the aim of supporting translational research. The availability of high
  • 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
  • EURO-NMD – An overview of European Reference Network for neuromuscular diseases

    A staggering 7000-8000 rare diseases affect the daily lives of around 30 million people across the European Union. Even though EU citizens benefit from improved access to healthcare throughout the
  • 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
  • Between hype and hope: the truth about gene therapy and genome editing in Duchenne muscular dystrophy

    About Duchenne muscular dystrophy Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy affecting around 1 in 5,000 newborn boys worldwide. The main characteristic is a
  • Meeting the partners — John Walton Muscular Dystrophy Research Centre

    Dr. Michela Guglieri is Honorary Consultant in Human Genetics and is working as a Neurologist and Senior Research Associate at the John Walton Muscular Dystrophy Research Centre (JWMDRC). Upon
  • Exercise in neuromuscular disorders: a moving topic

    Nicole Voet is a rehabilitation physician and scientific researcher in the Rehabilitation Centre Groot Klimmendaal in Arnhem and in the academic medical hospital Radboud University Medical Centre in