Ultragenyx Pharmaceutical RARE announced that the European Commission (EC) has expanded the eligible patient population for ...

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

Rising health awareness, increasing prevalence of various forms of mucopolysaccharidosis worldwide, continuous advances in ...

For example, if a child has coarse facial features and developmental delays, a pediatrician may have reason to believe that the child has a form of mucopolysaccharidosis. Mucopolysaccharidosis is ...

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 ...