Working in partnership

I think Muscular Dystrophy UK has done and is doing more than anyone in the UK to really try to pull the neuromuscular charities together, and I think this is admirable.

Professor Mary M Reilly MD FRCP FRCPI, Professor of Clinical Neurology and Consultant Neurologist, Queen Square, London

We are committed to building partnerships with other funding agencies and patient organisations. Combining our resources will make us more effective in funding and supporting the research that is needed.

We are grateful to all our partners for helping us to fund more high-quality research into muscle-wasting conditions.

The ENMC is an international organisation that supports research for neuromuscular conditions and strives to facilitate communication among scientists and clinicians working in the field. It funds and organises about eight workshops a year about neuromuscular conditions to encourage and facilitate collaborative research.

The workshops are chosen on the basis of scientific merit and each application is objectively assessed by the ENMC Research Committee, a group of internationally recognised experts in the field.

To date, the ENMC has organised around 200 workshops on a variety of neuromuscular disorders and published over 125 related publications. The workshops have been attended by over 1,600 participants from around the world.

The ENMC is funded by a group of patient and healthcare associations and pharmaceutical industries from around Europe, including Muscular Dystrophy UK. Our Director of Research, Dr Marita Pohlschmidt, is the current chair of the ENMC Executive Committee.

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TREAT-NMD is a global network for researchers and clinicians in the neuromuscular field. It promotes an infrastructure to ensure that potential treatments reach patients as quickly as possible. It brings together leading specialists, patient groups and industry representatives to promote best practice and ensure people are ready for the trials and therapies of the future.

Treat-NMD was launched in 2007 with the aim of ‘reshaping the research environment’ in the neuromuscular field. It has been instrumental in developing tools needed to bring new therapeutic approaches from the laboratory into the clinic.

Muscular Dystrophy UK has worked closely with this initiative to promote patient registries. We are currently supporting a registry for people with facioscapulohumeral muscular dystrophy (FSH) and myotonic dystrophy. We have also worked together to establish best practice care guidelines for people with a number of muscle-wasting conditions.

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Working with SMA Support UK (formerly the Jennifer Trust) and the SMA Trust, we jointly established SMA-REACH UK. This is a new information hub designed to improve and ensure consistency of healthcare for people living with spinal muscular atrophy (SMA). It will also pave the way for clinical trials into potential treatments for SMA, simplifying the co-ordination of research.

The hub will combine and build on information held in Newcastle University’s SMA Patient Registry (funded by SMA Support UK and Treat-NMD), and the SMArtNet database (backed by SMA Support UK and Muscular Dystrophy UK).

It will allow scientists and health professionals to share key information, including:

  • results of scientific research
  • knowledge of clinical practice
  • symptoms and disease progression
  • details of patients wishing to take part in clinical trials.

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In 2013, Muscular Dystrophy UK established the Duchenne Forum, a funding partnership between six UK charities dedicated to funding research into Duchenne muscular dystrophy. This collaboration between Muscular Dystrophy UK, Alex’s Wish, the Duchenne Children’s Trust, Duchenne Research Fund, Joining Jack and Harrison’s Fund will accelerate progress in the search for effective treatments and eventually cures.

The partnership is currently supporting nine pioneering research projects into Duchenne muscular dystrophy. In its first year, over £240,000 was spent on research through the Duchenne Forum. A further £800,000 will be spent from 2014 to 2018 on these projects.

By working in collaboration, the Duchenne Forum has been able to make a larger investment to move promising therapies forward. It also enables us to avoid duplication and keep administrative costs to a minimum.

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Muscular Dystrophy UK is working with SMA UK to co-fund two exciting, newly awarded research projects into spinal muscular atrophy (SMA). More than £200,000 is being committed to these projects at the universities of Edinburgh and Oxford over three years.

The aim of this partnership, established in 2014, is to accelerate progress in the search for effective treatments and eventually cures for the condition.

This partnership comes at a critical time, helping keep the momentum going now that the first potential therapies are in the pipeline. Researchers will be learning more about the underlying biological mechanisms of the condition and how best to optimise the effects of current and future therapies.

Co-funding these projects will help move promising therapies forward, avoiding administrative duplication and keeping costs to a minimum. The alliance is encouraging news for the thousands of families living with SMA who loyally support our charities.

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