Handy info for parents affected by Duchenne

If you are affected by Duchenne Muscular Dystrophy (DMD), you may find these links a useful source of information:

  • To view the latest list of active and upcoming clinical trials visit clinicaltrials.gov
  • Understanding Clinical Trials can be incredibly complex, we have found Duchenne UK’s site a useful guide
  • Understanding Clinical Research and the stages involved from getting an idea into clinic
  • It is so inspiring and helpful to read stories from those affected by Duchenne
  • If you need help and support, we have found Action Duchenne a fabulous place to start – they are in touch with so many parents, children and young adults affected by Duchenne
  • Every parent and carer looking after someone affected by Duchenne, would benefit hugely from reading the latest Standards of Care published by the Centers for Disease Control (CDC) in The Lancet Neurology:
  • We work with a number of charities in the UK who are also working on Duchenne Muscular Dystrophy, these include:
    • Duchenne UK – Alex’s Wish are a Partner Charity, set-up by two parents Emily Crossley and Alex Johnson both have children affected by Duchenne
    • Muscular Dystrophy UK – produce a number of useful factsheets and guides for those newly diagnosed, and for parents and caregivers
    • Action Duchenne – holds an annual conference each year in November where scientists from around the world are invited along to discuss progress being made on clinical trials, research projects and care standards (we highly recommend you attend this annual event if you are a parent or caregiver, it’s a great place to meet other parents affected by the condition)
  • The DMDRegistry is the first patient registry in the UK for Duchenne and one of the first neuromuscular conditions in the world. This patient registry has been developed by Action Duchenne and is a vital source of information when recruiting children onto clinical trials.  If you are a parent or carer to a child with Duchenne, be sure to register them as soon as possible. The UK DMD Registry is part of the TREAT-NMD Global DMD Registry.
  • The TREAT-NMD Alliance is an international network of clinicians, scientists, and patient organisations working with neuromuscular diseases including Duchenne muscular dystrophy.  It aims to help the most promising potential therapies reach patients as quickly possible by reducing barriers to ‘translational research’ – the process of going from a laboratory research to an effective treatment.