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

If approved by the EMA, mavorixafor would be the first drug indicated for patients with WHIM syndrome in Europe, a population estimated to be approximately 1,000 people. Earlier this month X4 ...

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).

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 ...

Tikun Therapeutics Inc. has obtained U.S. orphan drug and rare pediatric disease designations for its programs in familial dysautonomia, namely its rAAV2-U1a-hELP1 gene replacement therapy for the ...

Nuvalent, Inc. shares have fallen approximately 30% after promising updates on its two lead NSCLC candidates rallied the ...