The FDA has approved GRAFAPEX™ and fludarabine as a preparative regimen in acute myeloid leukemia or myelodysplastic syndrome ...

X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today announced that its Marketing Authorization Application (MAA) for ...

The FDA has granted orphan drug designation (ODD) to Zai Lab’s ZL-1310, aimed at treating small cell lung cancer (SCLC).

The goal is to treat familial dysautonomia patients with a single injection.

Intellia Therapeutics has dosed the first subject in the Phase III trial of NTLA-2002, to treat hereditary angioedema (HAE).

Stealth BioTherapeutics Announces PDUFA Action Date Extension for Elamipretide to Treat Patients with Barth Syndrome ...

The NPI-001 (N-acetylcysteine amide) tablets are a proprietary investigational therapy for the treatment of patients with ...

Zydus has announced that the USFDA has granted Orphan Drug Designation (ODD) to Usnoflast, a novel oral NLRP3 inhibitor, for ...

Zydus Lifesciences added 1.10% to Rs 987.60 after the company said that the USFDA had granted orphan drug designation (ODD) to Usnoflast, a novel oral NLRP3 inhibitor, for the treatment of amyotrophic ...

Zydus’ Usnoflast receives US FDA orphan drug designation to treat amyotrophic lateral sclerosis: Our Bureau, Mumbai Thursday, January 23, 2025, 12:15 Hrs [IST] Zydus, a leading, ...

Zai Lab Limited (ZLAB) announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation or ODD to ZL-1310, a ...

NDAQ:ZLAB) Zai Lab Receives Orphan Drug Designation from the U.S. FDA for ZL-1310 (DLL3 ADC) for the Treatment of Small Cell ...