What we’ve achieved

Impact Report 2020

We are focused on results. Everything we do is designed to improve the quality of life of people living with muscle-wasting conditions. We won’t stop until effective treatments and cures are found for all muscle-wasting conditions.

With your help we have already achieved so much:

  • We have invested more than £55 million in research using a robust international peer review process. We have laid the foundations for the first potential treatments for Duchenne muscular dystrophy and Becker muscular dystrophy.
  • In 2018/19, we invested more than £1.6m in research, as well as £180k directly to support clinical trial infrastructure, whilst in 2019/20 we funded nine new research grants bringing our portfolio to 50 projects into 14 different conditions – and we won’t stop until there are treatments and cures for all people living with muscle-wasting conditions.
  • MDUK-funded researchers published a paper showing they could correct a mutation commonly found to cause Ullrich congenital muscular dystrophy (UCMD), and further research could see the development of a potential treatment.
  • We funded research that led to a scientific breakthrough in the development of an IVF technique. It has the potential to prevent mitochondrial myopathy being passed from mother to child.
  • We announced a new artificial intelligence technology partnership with Healx to deliver treatments for facioscapulohumeral muscular dystrophy (FSHD). Healx uses AI technology to identify existing drugs to repurpose, combine and treat rare conditions.
  • The MDUK Oxford Neuromuscular Centre – a partnership between MDUK and the University of Oxford – is driving research into potential treatments from the lab to the clinic.
  • Working in partnership with individuals, families, clinicians and other patient groups we have secured NHS access to the Duchenne muscular dystrophy treatment Translarna and the spinal muscular atrophy treatment Spinraza.
  • We have been involved in the early stages for access to Namuscla for non-dystrophic myotonia, Raxone for Duchenne muscular dystrophy, and Zolgensma and risdiplam for spinal muscular atrophy.
  • Since 2013 we have secured 102 new neuromuscular specialist NHS roles.
  • Bringing families together to campaign with us, we have secured  £6.9m per year of NHS investment in specialist neuromuscular services across the UK.
  • In 2019/20, we helped around 985 people access the care and support services they are entitled to; securing £1.5m worth of benefits, services and equipment for people living with muscle wasting conditions
  • In 2019/20, 25,335 people understood more about their condition or got support from us online, over the phone or in person, and  more than 470 people called our Research Line to find out about the latest developments and trials.

We know we can beat muscle-wasting conditions more quickly by working together. Please join us.

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