Miracle drug plea for sick Hatfield girl
PUBLISHED: 14:09 03 October 2012 | UPDATED: 14:20 03 October 2012
A CLINCIAL drug trial may save a four-year-old girl from a rare degenerative disorder which will strip her of her movement and could her kill her before her 14th birthday - but her parents need to raise £600,000.
Tillie Mae Mawdsley was diagnosed with Sanfilippo syndrome two years ago and has already started to deteriorate.
But her decline could be halted if funding can be found to test miracle drug Genistien.
Her mother Michala, 32, said: “Tillie Mae is gradually losing her power of speech and she is very hyperactive, which is part of the condition.
“She has no fear of danger and is on the autistic spectrum which means she can become very withdrawn and go into her own little world.”
The Hatfield resident added: “I was devastated when she was diagnosed, just heartbroken.”
In order to secure the small fortune to pay for the clinical trial Mrs Mawdsley has clubbed together with other families and is working with new charity Wicked Genes, which is an off-shoot of The Society for Mucopolysaccharide Diseases.
The family aims to raise the cash by visiting schools to raise awareness and arrange fundraising events.
Although nothing has been organised yet it is hoped businesses will get behind the cause as well.
Mrs Mawdsley said: “It’s important. We need to get it available for Tillie Mae because they are going to trial it with children in a group situation.
“This drug is not going to cure them or repair the damage that has been done, it will hopefully halt the deterioration and improve their quality of life.”
The campaign has not begun in earnest but Tillie Mae, who attends Lakeside School, has been helped by her sister Lexie who has been “fantastic”.
“She describes Tillie Mae as having a poorly brain and says ‘are we going to go to hospital and get some medicine to fix her poorly brain’,” Mrs Mawdsley said.
To donate to log on to www.shareagift.com/giftpagefortillie1
More information on Wicked genes can be found online at www.wickedgenes.co.uk