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.
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).
http://www.actionduchenne.org/ 
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