The top line comprised sales of Galafold (migalastat), which is approved for Fabry disease and the newly approved combo drug, Pombiliti + Opfolda. The FDA approved Pombiliti + Opfolda, a two ...

which has filed a rolling marketing application for its AT-GAA (cipaglucosidase alfa/migalastat) candidate with the FDA. Amicus' drug also failed to show a significant improvement of Lumizyme in ...

"We closed 2024 beating expectations with exceptional revenue growth of 33 percent. With our portfolio and global rare disease capabilities, we have a clear path to deliver continued revenue growth ...

Since the National Institute for Health and Care Excellence (NICE) formally took on the appraisal of Highly Specialised Technologies (HSTs) for the rarest conditions in April 2013, just two drugs ...

The authors use deep mutational scanning to assess the effect of ~6,600 protein-coding variants in MC4R, a G protein coupled receptor associated with obesity. They develop new, more precise approaches ...

Laboratory of Computer-Aided Molecular Design, Division of Medicinal Chemistry, Otto-Loewi Research Center, Medical University of Graz, Neue Stiftingtalstr. 6/III, A-8010 Graz, Austria ...