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 ...

Some forms, like MPS I (Hurler syndrome) and MPS II, also involve severe neurological decline, while others, such as MPS IV (Morquio syndrome), primarily affect the skeletal system without ...

US biotech Regenxbio (Nasdaq: RGNX) has successfully completed the BLA submission for its mucopolysaccharidosis II (MPS II) ...

Regenxbio closes strategic partnership with Nippon Shinyaku for MPS diseases: Rockville, Maryland Wednesday, March 5, 2025, 14:00 Hrs [IST] Regenxbio Inc., a leading clinical-stag ...