Searching for a cure, Improving lives

Aims and Objectives

Where did we come from?  Started as Parent Project UK, our founders (Janet and Nick) laid out our vision:

 To be a leader in supporting innovative research to cure and treat Duchenne.

 To work as a partner with organisations providing resources to help to improve the length and Quality of Life of those living with Duchenne.

 To be led by parents and young people affected by Duchenne and Becker Muscular Dystrophy.

 This has evolved into four areas within which Action Duchenne now focuses:

Cure: To change Duchenne into a treatable and manageable condition by producing, and delivering, a broad research strategy that seeks to find the cure or treatment for Duchenne or Becker  muscular dystrophy.

Influence:  To inform political debate in the fight to improve the quality of life for those living with Duchenne or Becker muscular dystrophy by changing attitudes in standards of care, healthcare professionals and to influence the bench to bed times for treatments for those suffering with rare conditions.

Empower:   To continue to provide education for those living with duchenne/beckers muscular dystrophy through Decipha and Takin’ Charge by: continuing to fund Decipha to provide specialist learning assessments and interventions to support young people , their families and schools and to increase attendance at the International Conference.

Improve:   To make the DMD registry as the database of choice for pharma and clinicians by increasing the numbers of those registering on the database and to increase revenue from it.

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Events

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Registry

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Action Duchenne launches new Research Strategy for Duchenne muscular dystrophy

November 17th, 2017

  Action Duchenne, a leading UK-wide patient and parent-led organisation for Duchenne Muscular Dystrophy, is

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Long-term microdystrophin pre-clinical work, moving towards a potential therapy restores muscle function in canines

July 26th, 2017

Promising microdystrophin pre-clinical work, moving towards a potential therapy has shown to restore muscle function

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FDA new approach would reduce number of patients treated with placebo

December 11th, 2017

The U.S. Food and Drug Administration (FDA) has published its recommendation for a new approach

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Results announced from survey ‘Juggling care and daily life’

December 11th, 2017

The 17th October 2017 was an important date for the rare disease community.  The highlights of the

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Duchenne – How do you measure a life?

Living with Duchenne – an animation

Taking charge of Duchenne

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