The mucopolysaccharidosis market is witnessing a surge in innovation, driven by cutting-edge gene therapies, enzyme replacement treatments, and novel drug developments. The market is poised for ...

Regenxbio has entered into a strategic collaboration worth $810m with Japan-based Nippon Shinyaku to advance gene therapies targeting the rare metabolic disorder mucopolysaccharidosis (MPS).

Jan. 30, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced data from its RGX-121 (clemidsogene lanparvovec) program for the treatment of mucopolysaccharidosis type II (MPS II ...

For example, if a child has coarse facial features and developmental delays, a pediatrician may have reason to believe that the child has a form of mucopolysaccharidosis. Mucopolysaccharidosis is ...

REGENXBIO Inc. (Nasdaq: RGNX) today announced data from its RGX-121 (clemidsogene lanparvovec) program for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, will ...