Searching for a cure, Improving lives

TREAT-NMD Alliance

 

The TREAT-NMD Alliance (www.treat-nmd.eu) 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.

Patient registries are a very important tool in translational research.  When planning a clinical trial, it is important that eligible patients can be found and contacted quickly – particularly in DMD, where many therapies and trials are targeted at specific mutations.  With relatively few patients both eligible and willing to participate in any given trial, international cooperation is critically important.

The UK DMD Registry is part of the TREAT-NMD Global DMD Registry.  National DMD registries from 34 countries contribute a subset of their data into a single international registry.  This holds a standardised set of core clinical information, including full genetic diagnosis, for over 7,000 individuals.  This data is anonymous: personally identifiable information, such as names and addresses, are never uploaded to the Global Registry.  Any patients who might be eligible for a trial are instead contacted directly by curator of the UK DMD Registry.

By collecting information, including genetic diagnosis, about more patients than any one national registry could on its own, the Global DMD registry helps researchers plan effective clinical trials and other studies.  The UK DMD Registry is an important part of this effort.

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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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