The six films explore how novel therapies, precision medicine and innovative technologies are being used in paediatric clinical trials to help bring discoveries made in the lab to the patients who need them - from those affected by ultra-rare diseases through to common conditions that affect thousands of children.
One of the films focusses on precision medicine research taking place at the National Institute for Health Research (NIHR) GOSH Clinical Research Facility (CRF). Watch the film to find out how this vital research is helping develop more targeted and effective treatments for children like Lottie and Ashley who have a rare form of rickets called X-linked hypophosphatemia (XLH).
Dr William van’t Hoff, director of the NIHR GOSH CRF, who features in the film said “Previously people with XLH were treated with high doses of phosphate and vitamin D, but the clinical trial Lottie and Ashley took part in offered a new more targeted treatment. This kind of precision medicine, or designer medicine as its sometimes called, is helping us find more effective treatments for patients at GOSH and children across the world. It’s a really exciting time to be involved in research with children”.
Dr van’t Hoff also led the “Future of children’s research” films project at GOSH. Speaking of his motivation for producing the films he said: “Following the GOSH CRF’s recent NIHR funding award CRF, I was eager to showcase the range of globally leading children’s research taking place not just here but at Clinical Research Facilities around the country. There is a real sense of partnership and collaboration which, together, is fundamental to improving research and care for children.”
Meet Lottie and Ashley
Siblings Lottie and Ashley both have X-linked hypophosphatemia (XLH) – a rare genetic form of rickets which causes pain, bending of the bones and poor growth. They have been taking part in a clinical trial of a more targeted treatment for their condition.