Loughborough Echo

£60,000 grant for special research

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SILEBY charity Alex’s Wish, which raises money for research projects and clinical trials to find treatments for life-limiting disease Duchenne Muscular Dystrophy, has given a £60,000 grant to support an innovative gene therapy research project.

Emma Hallam, founder of Alex’s Wish and mum to Alex after whom the charity is named, said: “This is a hugely exciting time for the Duchenne community. Several companies are now testing an approach that uses a synthetic gene to replace the faulty dystrophin gene which causes Duchenne.

“This is known as gene transfer using microdystr­ophin or, more commonly, gene therapy. The companies are using viruses to deliver the therapy.

“The early data looks very promising but there are challenges in getting this treatment out to the entire Duchenne population, mainly because some patients will have pre-existing antibodies to ward off the viruses and so will not currently be able to have the treatment.

“Duchenne UK has granted £655,000 to a pre-clinical Duchenne program with Evox Therapeuti­cs, a company looking at using exosomes to deliver the gene without using a virus. Alex’s Wish contribute­d £60,000 to Duchenne UK. We are really excited to be supporting a project investigat­ing a new potential method of gene therapy delivery. If successful we will be nearer our ultimate aim of all Duchenne patients producing their own dystrophin.

“We will keep raising money and investing into research projects and clinical trials until we find successful treatments and a cure for all boys suffering from this horrible life-limiting disease. We won’t rest until we have succeeded and we believe this project takes us one step closer.”

To find our more about Alex’s Wish and how you can get involved, visit https://alexswish.co.uk/

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