Christine Lavery, from the Society for Mucopolysaccharide Diseases said: "This breakthrough brings long-awaited hope not only to affected families of one of the rarest diseases in the world ...

We eagerly anticipate the results of the trial and hope that this may ultimately result in a potential treatment for Sanfilippo disease Dr Brian Bigger The study was funded by charity The Society for ...

“Sanfilippo is an untreatable mucopolysaccharide (MPS) disease affecting one in 89,000 children in the United Kingdom,” said Dr Bigger, who is based in the School of Biomedicine. “Children with ...