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LifeArc and Action Medical Research announce commitment of over £1m to research into rare diseases in children

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LifeArc, a UK-based medical research charity, and children’s charity Action Medical Research, mark Rare Disease day 2021 by announcing a total commitment of over £1m for research in children’s rare diseases. This joint fund supports UK-based researchers to progress high-quality scientific projects that could potentially improve the lives of children living with a rare disease[i].


LifeArc and Action Medical Research have most recently committed £610,000 to support three academic research projects will help move beyond a basic understanding of disease mechanism into the development of an intervention that could deliver real-world benefits to infants, children and young people with rare disease. 


Dr Catriona Crombie, Head of LifeArc’s Philanthropic Fund said: “Our partnership with Action Medical Research demonstrates our focus on improving the lives of people living with rare diseases. Through our collaboration we can continue to fund high quality research into rare diseases in children and provide our expertise to translate science into vital new interventions.”


The three projects, funded through the 2020 funding round, aim to develop treatments or diagnostics in the areas of Duchenne muscular dystrophy (which causes relentless muscle wasting and weakening); hypertrophic cardiomyopathy (a major cause of sudden cardiac death in young people); and pyridoxine dependent epilepsy (a rare form of epilepsy which can be difficult to treat).


Sarah Moss, Communications Director at Action Medical Research said: “We’re really pleased to announce our continued working together with LifeArc; research in this area is so important to help the thousands of families who are dealing with the daily challenges that rare diseases bring.”


Notes to editors


The further investment of £610,000 is on top of joint funding for two research projects awarded in the 2019 funding round at a total of £499,000.


To date, LifeArc has awarded around £8m to support innovation in rare diseases. In addition, LifeArc has committed a further £8m to support delivery of new gene therapy treatments through a unique partnership with the MRC, the largest funder of gene therapy research in the UK. Here, LifeArc is one of three partners who are funding a network of cutting-edge facilities to enable academic progress their novel gene therapy research into early-stage clinical trials and bring a new generation of medicines in healthcare.


A disease is defined as rare in Europe when it affects fewer than 1 in 2,000 people. There are over 300 million people living with one or more of over 6,000 identified rare diseases around the world each supported by family, friends and a team of carers that make up the rare disease community.


i. Available at rarediseaseday.org/article/what-is-a-rare-disease. Accessed 26 February 2021



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Hannah Severyn

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