PERFORMANCE: A drug trial in which Sheffield Children’s NHS Foundation Trust was the largest site has been successful, with promising results for babies with severe hypophosphatasia, who normally die after six months.
The drug was developed in the USA then trialled across the world with Sheffield as the largest trial site. A statement said “nine of the 11 babies who took part are doing well”. Only 35 babies in the world are thought to have the genetic condition at any one time.
The drug, called asfotase alfa, was developed by bone research scientists in Boston and Montreal. Children have been injected with the drug three times a week and clear results emerged after around three to six months. Results were recently published in the leading medical journal the New England Journal of Medicine.
Professor Nicholas Bishop, at the FT and Sheffield University, said: “The long term effects of this drug are not yet known but the results we’ve had so far on this trial are undeniable. It’s saved and changed the lives of nine children who would otherwise have died after just a few months.”
See attached statement