Regenxbio (RGNX) announced the closing of its previously announced strategic partnership with Nippon Shinyaku. Under the terms of the ...

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 ...

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 ...

The designated rare diseases in the country include Gaucher Disease, Maple Syrup Urine Disease, Pompe Disease, Galactosemia, Phynelketonuria, Methylmalonic Acidemia, Urea Cycle Defects, Hurler ...

Denali Therapeutics Inc.’s DNLI share price has dipped by 10.43%, which has investors questioning if this is right time to buy.

REGENXBIO Inc.  today announced the closing of its previously announced strategic partnership with Nippon Shinyaku. Under the terms of the agreement, REGENXBIO and Nippon Shinyaku will develop and ...

for Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome and RGX-111 for Mucopolysaccharidosis I (MPS I), also known as Hurler syndrome in the United States and Asia. "RGX-121 and RGX ...

RGX-111, aimed at treating MPS I, known as Hurler syndrome, utilizes an AAV9 vector to deliver the IDUA gene to the central nervous system and has shown positive interim data from a Phase I/II ...

Rare Disease Day, observed on February 28th, aims to raise awareness about rare diseases and the challenges faced by patients ...

35-year-old All-Star infielder Anthony Rizzo is arguably the most notable name remaining in free agency ahead of Opening Day. The three-time All-Star became a free agent after the Yankees paid a $6 ...