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 ...

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

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

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

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 ...

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, ...

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

KalVista (KALV) announced that Japan’s Ministry of Health, Labour and Welfare, or MHLW, has granted sebetralstat orphan drug designation. The ...