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29 July 2016

First Duchenne Trial opens at Bristol children's hospital

Bristol Royal Hospital for Children has opened its first Duchenne Muscular Dystrophy trial, facilitated by Action Duchenne funding and with the support of experts and local family members living with the condition.



The early phase clinical trial in Duchenne on a novel investigational product, that could potentially treat most young people living with the condition, will be a major step forward for local families living in the South West and South Wales. Over 30 experts and family members attended the launch in Bristol, welcoming the announcement.  

The community gathering also saw the announcement of Action Duchenne's non-ambulatory call for research, targeted to those who are wheelchair-bound and wish to be involved in future trials that will improve the quality of their lives.

Aaron and Jack Ebanks, 19 and 16, living with Duchenne have decided to support the cause for non-ambulatory research and are leading their own event on the weekend, going from Bristol Royal Hospital for Children to Birmingham Children's Hospital in their wheelchairs. They departed from Bristol on Friday afternoon and finish in Birmingham on Monday afternoon.

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Aaron and Jack








Action Duchenne, the largest UK community-led Duchenne charity, has committed to funding the clinical research physiotherapist specialising in paediatric neuromuscular disorder for two years. This will increase access to innovative drugs and treatments for patients in the South West and South Wales, reducing the travel times for patients and their families by avoiding the need to travel out of region to access this care.

Action Duchenne, with the support of the Duchenne community, funds international state-of the art research in Duchenne and this is the first time a specific call has been announced in the UK for those who are older and no longer able to benefit from paediatric investigational compounds.

Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3,500 live male births (around 2500 people have Duchenne in the UK). group 3