Fourteen year old Ali Zaidi has been part of a clinical trial of a new enzyme replacement drug for five years. Ali has Morquio Syndrome, a rare inherited metabolic disorder which causes an abnormal storage of large and complex sugar molecules within the cells of the body.
This leads to an abnormal function of several systems in the body and predominantly affects the skeletal system. People with Morquio Syndrome have a severely short stature, abnormal bone structure, progressive breathing difficulties and reduced mobility.
Ali said: “I would definitely recommend other patients or parents to ask their child’s doctor or nurse about being part of a clinical trial. I can already feel the difference and the improvements that are happening within me.”