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 ...

MPS-IH, also called Hurler syndrome, and rarely live past the age of 10 when untreated. Current treatment options for MPS-IH include allogeneic hematopoietic stem cell transplant (HSCT ...

SEWARD, Neb. (KOLN) - A Seward fifth grader battling a rare disease known as MPS, or Hurler Syndrome, was honored by her classmates Thursday. Following a proclamation from Governor Jim Pillen ...

The study was published in the Nature journal Scientific Reports. Hurler syndrome, also known as mucopolysaccharidosis type I (MPS I), is a genetic disorder affecting newborns that leads to severe ...

or MPS II, and RGX-111 for Hurler syndrome, or MPS I. Under the deal, REGENXBIO (NASDAQ:RGNX) will receive $110M upfront and up to $700M in potential milestone payments, plus double-digit ...

Approximately 60 percent of children born with MPS-I have the most severe subtype, MPS-IH, also called Hurler syndrome, and rarely live past the age of 10 when untreated. Current treatment options for ...