First treatment funded through new pathway

A new pathway in Sydney Hospitals has allowed the first baby to be treated for a rare disease with a treatment just entering clinical trials overseas.

The Innovative Therapies Pathway was created by Dr Michelle Lorentzos, Medical Lead for Advanced Therapeutics at Sydney Children's Hospitals Network, to accelerate access to highly personalised therapies for children with rare, complex and life-threatening conditions.

"We are entering a new era where highly personalised therapies for ultra-rare diseases are increasingly possible," Lorentzos said. "This goes beyond a single medication or a single patient; it's about a new way of delivering innovative treatments to children."