Derbhile Dromey (Irish Medical Times) writes that some patient-led organisations, such as
Duchenne Ireland, are stimulating research into medical conditions.
Patient-led organisations are playing a powerful role in stimulating
research into rare diseases that would not otherwise attract funding.
Organisations representing people with Duchenne muscular dystrophy
(DMD), for example, which affects 160 families in Ireland, are
stimulating significant research into the condition. Duchenne Ireland
was set up by the parents of boys with DMD.
Though only in its infancy, it negotiated a deal through UK
organisation Action Duchenne to self-register with UK registries that
select subjects for clinical trials. It also secured an agreement from
the Tánaiste to facilitate a debate about DMD in the Dáil — a step towards permanent Government funding.
Duchenne Ireland gives an international focus to its research
efforts. It has created a €200,000 grant, entirely through its own
resources, which it has promoted through the research network Treat
Neuro-Muscular Disease (Treat-NMD). “We wanted to move things forward,”
said John Gorman of Duchenne Ireland (DI). “We don’t want an effort
where it’s research for research’s sake. A lot of research in Ireland
is university-led and may not be as translational as we’d like. We
wanted to concentrate directly on research, because we knew that
without that push, nothing was going to happen.”
The grant call is being peer-reviewed and DI has set up a research
committee of international researchers. “We’re looking at work where
questions linked to clinical trials will be answered. The key was to
make sure that it was as professional as possible. We’re not scientific
people, but we want to be facilitators. Everyone agrees that there may
not a cure or a therapy that will give function back to the boys, but
things can be done on a step-by-step basis that can improve their
quality of life. The research has to be applicable to this generation.”
Extract from Full Article kindly from Irish Medical Times