CURE13h
Bexobrutideg Receives Orphan Drug Designation in Waldenström Macroglobulinemia
Bexobrutideg, a bioavailable agent, was granted orphan drug designation by the FDA for Waldenström macroglobulinemia, a type ...
Zacks Small Cap Research on MSN14h
QNRX: With Clinical Activities Expanding, QNRX Expects Cash Runway Into 2Q26
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 ...
CURE17h
FDA Grants Orphan Drug Designation to PEP-010 in Pancreatic Cancer
The FDA has granted orphan drug designation to PEP-010 for pancreatic cancer, advancing its development as a potential novel ...
The Manila Times21h
Nurix Announces U.S. FDA Orphan Drug Designation Granted to Bexobrutideg (NX-5948) for the Treatment of Waldenström Macroglobulinemia
Orphan Drug Designation follows positive Phase 1 data presented at the 12th International Workshop on Waldenström ...
inews.co.uk on MSN1d
The 3.5m people with rare diseases who face worsening treatment and diagnosis rates
Rare diseases affect one in 17 people in the UK, but a Government initiative designed to improve their quality of life will ...
For kids with extremely rare diseases, this promising technology provides a lifeline | Opinion
Genetically targeted technologies are built on Nobel Prize-winning science. They represent a scientific “quantum leap” that ...
American Council on Science and Health3d
“Rare” but Not Uncommon: The Hidden Crisis of Rare Diseases
Rare diseases - a medical condition so obscure that even your doctor has to Google. Despite their name, these diseases ...
Korro Bio, Inc.: KRRO-110 Receives Orphan Drug Designation from U.S. FDA for Alpha-1 Antitrypsin Deficiency
Korro Bio, Inc. (Korro) (Nasdaq: KRRO), a clinical-stage biopharmaceutical company focused on developing a new class of genetic medicines based on ...
Korro Bio gets Orphan Drug Designation from FDA for its genetic disorder treatment
Korro Bio received U.S. FDA's orphan drug designation for investigational medicine KRRO-110 to treat Alpha-1 Antitrypsin ...
The Manila Times3d
KRRO-110 Receives Orphan Drug Designation from U.S. FDA for Alpha-1 Antitrypsin Deficiency
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, Inc.: Quoin Pharmaceuticals Provides Corporate Update and Reports Fourth Quarter and Full-Year 2024 Financial Results
Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the "Company" or "Quoin"), a late clinical-stage specialty pharmaceutical company focused on rare and orphan ...
Healio5d
FDA grants fast track designation to X-linked retinoschisis treatment
The FDA granted fast track designation to ATSN-201 for the treatment of X-linked retinoschisis, according to a press release ...