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 ...

or Hurler-Scheie syndrome, is a rare lysosomal storage disorder caused by the inability of the body’s cells to produce enough of an enzyme called alpha-iduronidase that breaks down ...

In 2021, Gunner from Highley, Shropshire, was diagnosed with Mucopolysaccharidosis Type I Hurler Syndrome ... including enzyme infusions, and a previous stem cell transplant, which failed.