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

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

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 ...

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 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).

Stealth BioTherapeutics Inc. (the "Company" or "Stealth"), a clinical-stage biotechnology company focused on the discovery, ...

Ahmedabad: Zydus, a discovery-based, global pharmaceutical company has announced that the U.S. Food and Drug Administration ...

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

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

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, ...

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