Ten presentations at ESGCT 2024 detail the therapeutic ... portfolio in metachromatic leukodystrophy (MLD), the Hurler subtype of mucopolysaccharidosis type I (MPS-IH), and mucopolysaccharidosis ...

The first presentation highlights non-clinical ... aimed at treating rare diseases including MPS I (Hurler, Hurler-Scheie and Scheie syndrome), MPS II, MPS IIIA and B (Sanfilippo syndrome type ...

the WORLDSymposium™ 2024 scientific program featured presentations focusing on: 52-week interim data from the JR-171 phase I/II clinical trial (Hurler syndrome, Hurler-Scheie syndrome or Scheie ...

Patients with the disease, called Hurler syndrome, suffer from short stature, spinal defects, and extremely stiff joints, complications that greatly limit their quality of life. But in a new paper ...

Mucopolysaccharidosis type 1, also known as Hurler syndrome, is a rare but life-threatening genetic condition. Babies born with this inherited disorder do not produce an enzyme needed to break down ...

The findings suggest the use of gene therapies—an entirely new standard for treatment—for those with brain disorders like Hurler syndrome, which have a devastating impact on those affected.

HURLER's syndrome is a genetically transmitted disorder of mucopolysaccharide metabolism characterized by excessive visceral storage of chondroitin sulfate B and heparitin sulfate, 1 and excessive ...

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 ...