DOCTORS in Sheffield have helped develop a new drug that could help save the lives of babies dying from a rare genetic bone disease.
Children with severe hypophosphatasia, which affects just 35 babies worldwide, began a clinical trial three years ago at Sheffield Children’s Hospital and other hospitals across the world.
The results of the trial have now been published, showing the asfotase alfa drug had been successful.
Experts from Sheffield Children’s Hospital and the University of Sheffield said it was ‘crucial’ the drug is licensed as soon as possible.
Nine babies completed the global trial, three of whom were given the drug in Sheffield.
Sufferers of severe hypophosphatasia normally die after the age of six months. Children struggle to walk or use their limbs properly, are very weak and in pain.
Prof Nicholas Bishop, an expert in metabolic bone disease based at Sheffield Children Hospital 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.”
Prof Bishop, who has been developing Sheffield’s bone service for the last 13 years, added: “The trial has been a fantastic success and it’s crucial we now get the drug licensed as soon as possible and begin helping children born with this disease.”