New role to help promote Internationally agreed Standards of Care for Duchenne Muscular Dystrophy
Action Duchenne today announced the appointment of Dr. Karen Rafferty as TREAT-Duchenne UK Coordinator. Dr. Rafferty will be working with TREAT-NMD and Action Duchenne on a range of duties, which will include managing the dissemination of the soon to be announced internationally agreed Standards of Care for Duchenne Muscular Dystrophy to clinicians across the UK, and working with clinical teams in the UK to help to establish trial ready sites to support new trials for Duchenne medicines. In addition, Dr. Rafferty will be working with the DMD registry, setting up a steering committee and developing terms of reference with the long term goal of integrating the UK registry with the Global TREAT-NMD registry. The post has been funded by the charity Action Duchenne.
The dissemination of the Standards of Care for Duchenne will include a ‘family friendly’ booklet, which will explain the range of care recommended for each aspect of the condition in easy to understand language. Once written the booklet will be available for download from the Action Duchenne and TREAT-NMD websites. Dr. Rafferty will also be working to promote the Accident & Emergency Care Pack for Duchenne patients, ensuring that clinicians and families know about the pack. Patients and clinicians will be able to register for a pack online at the Action Duchenne website, and the pack will either be sent, or can be downloaded.
Dr. Rafferty has a Masters degree in Medicinal Chemistry and for her PhD she researched ‘Novel Transition Metal Complexes as Potential Anti-tumour Agents.’ Dr. Rafferty has been based in Ireland during the last year, working as an academic editor for Compuscript Ireland and as a Technology Transfer Project Manager at NUI Galway.
Dr. Rafferty commented, “I am excited about the possibilities of this new post. Having a background in bench chemistry and producing drugs I am keen to see the other end of the spectrum where drugs are going into clinical trials. As TREAT-Duchenne UK Coordinator my project management skills will be key to making the post a success and I’m looking forward to seeing the Standards of Care implemented and making a real difference to the lives of children and young men with Duchenne.”
Nick Catlin, CEO of Action Duchenne said; “We are delighted with Dr. Rafferty’s appointment. Her work will be key in helping to ensure that all patients and families in the UK receive comparable care for what is a very complex condition, ending the current postcode lottery for care.”
Duchenne muscular dystrophy is the most severe form of the condition, affecting around 3,500 people in the UK and about 40,000 people worldwide. It has no cure and is caused by mutations in the dystrophin gene that prevent correct reading of the genetic code, the result of which is that the essential muscle protein dystrophin is not produced. This results in progressive muscle wasting, severely limiting mobility in teenage patients (boys and men) and leading to premature death in their twenties from respiratory or cardiac problems (the heart muscle is directly affected by the genetic defect).
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About Action Duchenne
Action Duchenne (formally Parent Project UK) was set up by Duchenne families in 2001 to promote new research for a cure for Duchenne. The charity has a strong record in funding research and has to date funded 8 major projects costing £800,000 and has lead the £1.6m DoH funding of the MDEX project. These projects have enabled much needed early work to be completed on exon skipping and other therapeutic approaches.
Action Duchenne holds an international conference every year to bring together researchers and families to exchange new research developments and provide a vital meeting venue for scientists.
In 2005 Action Duchenne launched the Duchenne Registry, the first National Duchenne database that holds gene information of people living with Duchenne and can be used to speed up the recruitment of patients for clinical trials.
In 2006 Action Duchenne launched a comprehensive learning and behaviour toolkit for use by parents and education professionals.
For more information please visit: www.actionduchenne.org
CEO, Action Duchenne
Tel: 0208 556 9955
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Tel: 01491 639500
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