Orphan drugs offer critical treatment options for rare diseases, improving quality of life for underserved patients and ...

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

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

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

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

The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Zai Lab’s delta-like ligand 3 (DLL3) antibody-drug conjugate (ADC), ZL-1310, for small cell lung cancer (SCLC).

Zydus Lifesciences announced that Usnoflast, a treatment for amyotrophic lateral sclerosis (ALS), has received Orphan Drug Designation from the US FDA. This designation supports the development of ...

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

Zydus Lifesciences announced that the USFDA granted Orphan Drug Designation to Usnoflast, a novel oral NLRP3 inhibitor, for ...

CAMBRIDGE, Mass. & SALISBURY, England - KalVista Pharmaceuticals, Inc. (NASDAQ: NASDAQ:KALV) has received Orphan Drug ...

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, ...

Oppenheimer reaffirmed its positive stance on Catalyst Pharmaceutical (TADAWUL:2070) Partners (NASDAQ:CPRX), maintaining an ...