Blow for Welwyn Garden City family of seriously ill eight-year-old girl Tillie Mae

PUBLISHED: 14:51 13 October 2016 | UPDATED: 14:51 13 October 2016

Tillie, Michala, Lexie and Paul.

Tillie, Michala, Lexie and Paul.

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Relatives of a Hatfield girl who suffers from a rare disease have not given up hope after a drug trial was terminated.

Tillie Mae Mawdsley, eight, was diagnosed with Sanfilippo syndrome in 2010 when she was two years old and her family has been fundraising ever since.

Sanfilippo syndrome is a progressive disorder affecting the brain and causing developmental delay, severe hyperactivity, motor dysfunction, and death at a young age.

There is currently no cure.

In 2011 Tillie Mae went on a clinical trial run by Shire, an American pharmaceutical company, but this came to an immediate end on August 12, this year.

The Mawdsley family, of Heathcote Aveune, were left devastated but have refused to give up hope and are now raising money so they are prepared if another opportunity for another clinical trial becomes available.

Tillie Mae’s mum Michala, 36, said: “We truly believed that the trial drug would have been successful but we have not lost hope and I won’t ever lose hope.

“When Tillie Mae’s sister Lexie found out that the medicine had stopped she said ‘Tillie Mae is going to die now’, it was awful hearing it.”

Michala added: “Despite everything she is the happiest little girl.”

The Mawdsley family are now aiming to get Tillie Mae on one of two gene therapy trials currently taking place in America and France.

Michala said: “We don’t know if we will meet the criteria or even if it will become available in the UK but we have to try. What else as parents can we do?”

To follow Tillie Mae’s story go to ‘Princess Tillie Mae fighting Sanfilippo’ on Facebook.

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