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

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

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

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

Dublin, Jan. 22, 2025 (GLOBE NEWSWIRE) -- The "Orphan Drugs - Global Strategic Business Report" report has been added to ResearchAndMarkets.com's offering. The global market for Orphan Drugs was ...

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

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 ...

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

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).

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

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