Bexobrutideg, a bioavailable agent, was granted orphan drug designation by the FDA for Waldenström macroglobulinemia, a type ...

BioArctic AB (publ) (Nasdaq Stockholm: BIOA B) announced today that the US FDA Office of Orphan Products Development (OOPD) has granted orphan drug ...

QNRX READ THE FULL QNRX RESEARCH REPORT Progressing studies of QRX003; positive interim clinical data Quoin Pharmaceuticals (NASDAQ:QNRX), a clinical stage, specialty pharmaceutical company that ...

The FDA has granted orphan drug designation to PEP-010 for pancreatic cancer, advancing its development as a potential novel ...

Orphan Drug Designation follows positive Phase 1 data presented at the 12th International Workshop on Waldenström ...

Rare diseases affect one in 17 people in the UK, but a Government initiative designed to improve their quality of life will ...

Genetically targeted technologies are built on Nobel Prize-winning science. They represent a scientific “quantum leap” that ...

Rare diseases - a medical condition so obscure that even your doctor has to Google. Despite their name, these diseases ...

Korro Bio received U.S. FDA's orphan drug designation for investigational medicine KRRO-110 to treat Alpha-1 Antitrypsin ...

KRRO-110 is the first RNA editing development candidate from Korro's proprietary OPERA TM platform and is currently being evaluated in the Phase 1/2a REWRITE clinical study for AATD. Dosing of the ...

Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the "Company" or "Quoin"), a late clinical-stage specialty pharmaceutical company focused on rare and orphan ...

Today announced strategic collaboration with Hologen AI, including a $200 million upfront payment to MeiraGTx and the formation of a joint ...