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.”

International researchers

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