Regenxbio will receive $110m upfront from Nippon Shinyaku and is eligible to get up to $700m in development, regulatory, and ...

Some sequence variants also cause inherited diseases, including sickle cell anemia, cystic fibrosis, and mucopolysaccharidosis type III. However, it is often difficult for scientists to identify which ...

REGENXBIO's partnership with Nippon Shinyaku provides $110M upfront, ensuring a cash runway for gene therapy programs in MPS ...

Nanosonics (ASX:NAN) has achieved a feather duster-to-rooster transformation with a bullish trading update for the December ...

The second asset, RGX-111, is being developed for mucopolysaccharidosis type I (MPS I), caused ... Plans are also underway for a Phase III study of the therapy in diabetic retinopathy. Additionally, ...

As the year unfolds the biotech industry will look closely at these ten first-in-class drugs on track to reach FDA approval ...

MPS III was the type for which neurological signs and symptoms were most frequently reported at presentation. Physician survey: signs/symptoms present when mucopolysaccharidosis (MPS) first suspected ...

Its commercial products include Vimizim, an enzyme replacement therapy for the treatment of mucopolysaccharidosis (MPS) IV type A, a lysosomal storage disorder; Naglazyme, a recombinant form of ...

Martin Luther King III, the late civil rights leader's son and a longtime activist himself, spoke with CBS News the morning of President-elect Donald Trump's second inauguration, touching on how ...