Research to Tackle Charlie Gard's Condition


New research into a therapy for the currently untreatable mitochondrial depletion syndrome is to be funded by a UK charity. The condition was brought into sharp focus by the case of Charlie Gard, who died a week before his first birthday as his parents fought to allow him to be treated in the US with a new form of treatment called nucleoside therapy.


Liz Curtis, founder of the Lily Foundation which will help fund the research, says it could provide the help families like hers so desperately need. Her daughter Lily died at 8 months.


“Currently there are no treatments, and there is no cure for mitochondrial disease,” she said. “So we need to keep looking to find something that will improve the quality of life for the families and the children who are suffering.”


The research, jointly funded by the Australian Mitochondrial Disease Foundation, will be undertaken at the Wellcome Centre for Mitochondrial Research in Newcastle.


Professor Robert McFarland from the centre hopes that it could expand the effectiveness of the therapy. What’s available at the moment is very limited for a very specific form of mitochondrial DNA depletion syndrome, he said. “What we hope is the molecules that we are developing and testing in these cells will be much more effective at doing this job of repleting mitochondrial DNA and that we will be able to look at outcome measures in patients that will tell us if it’s been effective.”

Sky News. June 24.

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