A five year-old boy with a life-limiting metabolic disorder is celebrating Christmas, after a vital stem cell transplant. In ...

Data from the first patients enrolled into Regenxbio's trial of its gene therapy for rare inherited disease mucopolysaccharidosis type I (MPS I) – also known as Hurler syndrome – has shown the ...

In 2021, Gunner from Highley, Shropshire, was diagnosed with Mucopolysaccharidosis Type I Hurler Syndrome, which stops his body breaking down sugar. He was given one-to-two years to live without a ...