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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Summit presents data from Phase 1 clinical programme of lead utrophin modulator, ezutromid

June 22nd, 2017

Summit Therapeutics has today announced the presentation of clinical data from two Phase 1 clinical trials

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What Raxone being given approval through the EAMS scheme means for the Duchenne community

June 22nd, 2017

  Over a number of years we have worked closely with Santhera Pharmaceuticals and other

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