Searching for a cure, Improving lives

Campaign Objectives

Action Duchenne are campaigning to ensure:

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1. All children and adults living with Duchenne have opportunities to participate in clinical research and access high quality multidisciplinary care

Research into Duchenne has reached an unprecedented stage, with a tidal wave of emerging treatments now in the pipeline. However, if these are to be developed it is essential the UK has the capacity to host clinical trails, capable of linking patients with innovative emerging drugs and proving the clinical efficacy of said treatments.

Our ‘Joining the Dots’ initiative and partnership in the ‘Newcastle Plan’ therefore aims to develop the capacity of the UK to host clinical trials and ensure every person living with Duchenne in the UK has access to clinical research opportunities and the highest standards of multidisciplinary care.

 

2. Safe and effective treatments are made available to those living with Duchenne without delay.

In the summer of 2014, Translarna (Ataluren) became the first European Medicines Agency approved treatment to address the underlying causes of Duchenne muscular dystrophy. Since this date we have campaigned tirelessly to ensure this treatment receives funding approval and is made available to all those eligible to benefit.

We are also fighting to ensure that the UK has a quick, robust, transparent and fair process for evaluating future Duchenne treatments, and to guarantee the mistakes, misguidance and inertia displayed in NICE and NHS England in their evaluation of Translarna, cannot be repeated. These efforts extend to focussing upon the regulatory framework of clinical trial and drug approval, encouraging funding for Duchenne research and the development of improved and adaptive regulatory pathways.

 

3. Everyone living with Duchenne can lead a long a fulfilled adult life 

Despite huge advances in research and the clinical management of Duchenne, a collective failure to acknowledge the potential of every child living with Duchenne has led to many living unfulfilled lives in adulthood.

In addition raising to expectations amongst patients and families, we further inform health, education and social care professionals about the learning and behavioural difficulties associated with Duchenne, and the need for good educational outcomes as a basis for a well rounded adult life.

 

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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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