2,000 Lost Boys with Duchenne Muscular Dystrophy

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VASTox plc are supporting the Duchenne Muscular Dystrophy Charity PPUK in developing a new Registry to accelerate clinical trials that might lead to new treatments. But PPUK has still to make contact with some 2,000 UK families who will benefit from involvement in the Duchenne Muscular Dystrophy Registry.

Parent Project UK is pleased to announce that VASTox plc, a leading chemical genomics company, announces today it has provided a £20,000 grant to the UK muscular dystrophy charity. The purpose of the grant is to support the management and development of PPUK’s DMD Registry (http://www.dmdregistry.org).

The DMD Registry is a national DMD database for clinicians, health professionals and researchers to help accelerate the development and delivery of new treatments for Duchenne and Becker muscular dystrophies (DMD and BMD) by providing an easier path to clinical trials in the UK.

DMD is a severe genetic muscle wasting disease that will mean boys will be in wheelchairs by 10 and without treatment will die in their late teens.

However, since the launch of the Registry in October 2005 only 100 families who have sons with DMD have so far registered. This could mean that some 2,000 boys and their families in the UK with Duchenne have not heard about the Registry.

It is vital that PPUK finds these families as soon as possible. Ultimately, this registry will be used by VASTox, and other organisations engaged in the development of therapies for muscular dystrophies, to support Phase II and Phase III clinical trials in the UK. By registering families will be given up to date information on new potential trials of treatments for DMD. In some cases they might be asked to be involved in new clinical research.

Nick Catlin, CEO of PPUK, said, “Duchenne Muscular Dystrophy is a devastating severe muscle wasting disease. Most families are told that there is no hope for a cure or treatments for DMD. This results in the terrible prospect of waiting for your son to die. VASTox is leading the way in the UK by developing new drugs in the future that might give our sons a longer and better quality of life. Parents of boys with DMD must understand that we all have a very important part to play in helping to accelerate this research. By registering with the DMD Registry we will give companies like VASTox access to a world-class, streamlined clinical trials system. This grant from VASTox is wonderful and will help us to be able reach more families so that we can provide the essential clinical and gene variation information on patients for clinical trials that will help us in our race against time to treat DMD."

Steven Lee, CEO of VASTox said, ”We are very pleased to provide a grant to the PPUK for this worthy project. For VASTox, the DMD Registry will be very useful in preparing and recruiting for the clinical trials of our lead DMD drug development programme. But more widely, this resource will be extremely valuable for everyone involved in the search for an effective therapy for DMD. We are looking forward to working more closely with PPUK in the coming months.”

The grant will be used by PPUK to recruit a full-time project manager and to help raise awareness of the registry among clinicians, patients and their families.

The resource will also provide information for families of DMD patients, including the latest developments in research for treatments for DMD, which will enable them to make better decisions about their future medical care needs.

The registry aims to register every person in the UK with DMD and BMD, including detailed clinical and genetic data, in a secure, legally protected database that has been endorsed by senior clinicians, health professionals and researchers.

For more information please contact:

Parent Project UK (PPUK)

Nick Catlin, Chief Executive Officer

Tel: +44 (0)208 556 9955


Steven Lee PhD Chief Executive Officer

Darren Millington Chief Financial Officer

Tel: +44(0)235 443910

Citigate Dewe Rogerson

David Dible / Mark Swallow / Valerie Auffray    Tel: +44 (0)207 638 9571

About Parent Project UK:

Parent Project UK Muscular Dystrophy (PPUK) is the only national charity that exclusively funds research and campaigns for better medical care for Duchenne and Becker muscular dystrophy. PPUK was set up by parents of boys with Duchenne muscular dystrophy in 2001 and has since been instrumental in setting up a consortium of researchers to develop the first clinical trial for a Gene Therapy in the UK. The MDEX consortium has already won £1.6m of funding from the Department of Health following the lobbying of parents and supporters from PPUK. PPUK has networks of Parent Action Groups across the UK and has recently set up a DMD Registry, http://www.dmdregistry.org, that will be the first national database of all those living with DMD in the UK. PPUK will make this data available to help to accelerate future research and clinical trials.

Further information is available at http://www.ppuk.org

About VASTox plc:

VASTox is a chemical genomics technology company that discovers and develops proprietary novel drugs and provides services to the pharmaceutical industry. The company’s most advanced drug development programme is focused on developing a new treatment for Duchenne muscular dystrophy based on the up-regulation of utrophin. A second drug development programme for spinal muscular atrophy is also progressing rapidly. VASTox has three additional programmes focused on osteoarthritis, cancer and tuberculosis that are expected to be out-licensed prior to entering the clinic.

The company’s technology platform, which uses zebrafish and fruitflies, has the potential to dramatically decrease the time and cost of drug discovery and development. This is because using whole organisms allows it to carry out high volume, high content screening that delivers data which is highly predictive of the efficacy and toxicity of potential drug compounds in humans. VASTox is growing revenues based on marketing its unique technology platform and its chemistry expertise. The company listed on the AIM market of the London Stock Exchange in October 2004.

Further information about the company is available at vastox.com.


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