If you have been following Harley’s story, you will know that he has recently been diagnosed with MPS III, Sanfilippo Disease. This rare and life-limiting, genetic disease for which there is currently no cure, is caused by the body’s inability to produce specific enzymes.
Harley is now set to partake in a new clinical trial at Great Ormond Street Hospital, London. The trial will be funded by Pharmaceutical company BioMarin who provides breakthrough treatments to patients around the world suffering from rare genetic diseases.
Harley has been selected as one of only 3 children worldwide who are involved in the trial and the only child from the UK, at just 4 years old he is the youngest participant. This came about after Harley’s parents, Clark & Partners Lead Service Engineer, Wayne and Emma, trawled the internet for answers following their sons diagnosis. They communicated with an American father of MPS sufferer on the MPS Sanfilippo Disease Facebook page and this in-turn lead to communication with a clinical trial officer based in the UK.
The trial will initially involve Harley and his family making regular 6-weekly visits to GOSH, where his progress will be monitored for a 12 month period followed by weekly visits where treatment can be administered and scans, tests and various other invasive procedures carried out. Harley will need to visit GOSH every week for the following 3 years.
BioMarin will also support the family by funding accommodation and expenses while Harley is in London being monitored and undergoing treatment. So far the transport costs have been covered by East Midland Trains after a family friend employed there, highlighted Harley’s story to management.
Wayne said ‘The support we are receiving is so greatly appreciated, it enables myself and Emma to remain focused on Harley and his future without worrying about the additional financial implications of his treatment. It is hoped that this research will help slow down the progression of MPS and allow researchers more time to help find a permanent cure for our son, one that can save hundreds of lives every year.’
If you would like to donate to the MPS Society and read Harley’s story, please visit the JustGiving page: https://www.justgiving.com/fundraising/paul-williamson16
Find out more about the MPS Society here.