Promising Mucopolysaccharidosis Type I Pipeline Therapies such as Laronidase, Antihistamine, Antipyretic, JR-171, AGT-181, ...

BioMarin has been resetting its business with cuts in headcount and a narrowing of active programmes over the last year or so, but is now looking to the future with a takeover deal for Inozyme Pharma ...

In May 2025, Denali Therapeutics Inc. announced a Phase 2/3 study of the efficacy and safety of tividenofusp alfa (DNL310), ...

To help raise awareness about MPS, Kingston City Hall and Springer Market Square will be lit up in blue and yellow on May 15.

Adhiraj Gaikwad, a two-year-old with two rare genetic disorders, received a bone marrow transplant in Mumbai to treat Mucopolysaccharidosis Type 1 (MP ...

The parents of a five-year-old girl diagnosed with a rare form of childhood dementia have told of their heartbreak. Stuart ...

US FDA accepts and grants priority review status to Regenxbio’s BLA for clemidsogene lanparvovec to treat Mucopolysaccharidosis II: Rockville, Maryland Thursday, May 15, 2025, 1 ...

Stuart and Pam said they were "devastated" when doctors told them Tilly has MucoPolySaccharidosis (MPS3A) or Sanfilippo Syndrome - a genetic condition affecting the cells in the brain. It has no cure.

A Seward fifth grader battling a rare disease known as MPS, or Hurler Syndrome, was honored by her classmates Thursday.