Alex’s Wish pledges £20,000 to Duchenne UK’s gene therapy grant call

August 30, 2022 1:27 pm

Alex’s Wish has pledged £20,000 so far towards a £1 million grant call into treatments which will ultimately allow everyone living with Duchenne Muscular Dystrophy to receive gene therapy safely and effectively.  Alex’s Wish are also hoping to pledge another £80,000 over the early Autumn months to support this project from their fundraising activities over the Summer and Autumn 2022.

The Leicestershire-based charity is supporting national organisation Duchenne UK to build on the success of clinical trials into therapeutic treatments for Duchenne, with the first trials coming to the UK last year.

Duchenne is a serious muscle-wasting disease affecting one in every 3,500 boys for which there is currently no cure.

Emma Hallam, CEO of Alex’s Wish, and mum to teenager Alex who lives with Duchenne, said there had been incredible progress in gene therapy for Duchenne, but challenges still lay ahead.

Emma said: “One of these challenges involves looking at how the immune system in some patients reacts to gene therapies. Some people have antibodies to the gene therapy viral vector, the vessel in which the genetic material is delivered to muscles, so they’re ineligible for this treatment.

In addition, even those patients without antibodies will develop them after receiving gene therapy, which means that they can’t have the treatment more than once.

We are however, now seeing novel, promising approaches, leading the way for cell-based therapies and new gene therapies that use non-viral vectors. Alex’s Wish is supporting this vital research, helping to accelerate its progress so that one day everyone with Duchenne can have treatments safely and effectively.

What we ultimately hope to see from Gene Therapy is that it will halt disease progression from the point that is taken. This will have a huge impact on all those affected by this condition.”

Alex’s Wish has launched a packed programme of exciting events throughout the summer and autumn months and will be using these as a springboard to raise funds and spread the message about ongoing medical research into Gene Therapy for those living with Duchenne.

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