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 ...

also known as Hunter syndrome, and RGX-111 for Mucopolysaccharidosis I, also known as Hurler syndrome. Regenxbio will receive $110 million and up to an additional $700 million if certain ...

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 ...

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 royalties on net ...