A two-year Daily Express campaign helped sufferers to secure wonder drug sapropterin on the NHS. The drug works for around 40 percent of the UK’s 2,300 PKU patients, enabling them to eat a normal diet.
Today sufferers, families and experts meet at the National Society for the Phenylketonuria (NSPKU) annual conference to hear how scientists are working on future treatments for those not suitable for sapropterin.
The Daily Express has vowed to keep fighting for them.
Olivia and Ruby, of Staple Hill, Gloucestershire, only started taking sapropterin on August 26, but it has already boosted their health, diet and mental wellbeing.
Mum Leanne, 46, said: “The girls are happier, enjoying their food, and healthier. It’s been an amazing transformation.”
“We are so lucky they respond so well to the drug and my husband Peter and I are thankful to all who helped them get it.”