Fresh hope for Hatfield girl in ‘wonder drug’ campaign
PUBLISHED: 17:00 13 July 2013
A HATFIELD girl who suffers from a painful disorder which limits her ability to move, could be chosen for a clinical trial after an epic fundraising drive.
Tillie Mae Mawdsley, five, from Hatfield, has a rare disease called Sanfilippo syndrome, a disorder which sees her joints stiffen.
Her family has been spearheading fundraising to pay for trials of a ‘wonder drug’, which is not available on the NHS, called Genistien.
In order to set up a trial mum Michala and her husband, Paul, were told they would need to raise more than £600,000.
The couple have already raised more than £170,000. Other families throughout the UK have also been donating money.
Enough money has been raised for preliminary trials to begin, but a small fortune still needs to raised to fund the full trials.
Michala, 32, said: “It is good, obviously, that it will start, but we are quite wary as there is still £200,000 that needs to be raised to carry out the full trial.
“It is difficult to say [how we feel] as we do not know much about it at the moment.”
The first part of the trial is due to start in the autumn at the University of Manchester, and there is a possibility Tillie Mae could be asked to take part.
Michala said: “This drug is not going to cure them or repair the damage that has been done, it will hopefully halt the deterioration and improve quality of life.”
Currently there is no cure for the life threatening illness.
■ To donate towards the clinical trial visit www.shareagift.com/giftpagefortillie1
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