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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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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First draft of agenda announced – Action Duchenne International Conference

June 28th, 2017

We are extremely excited to announce the first draft of the agenda for the Action

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Duchenne – How do you measure a life?

Living with Duchenne – an animation

Taking charge of Duchenne

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