The Food and Drug Administration (FDA) has granted Orphan Drug designation to BA-102 for the treatment of Phelan-McDermid syndrome, a rare neurodevelopmental disorder linked to autism.

NS Pharma, a unit of Nippon Shinyaku (OTCPK:NPNKF), said on Monday that the U.S. FDA has granted orphan drug designation to ...

NS-229 is an investigational selective Janus kinase 1 inhibitor designed to prevent tissue damage caused by EGPA by suppressing immune cell activation.

The FDA designation marks a significant milestone in the development of a novel treatment for a rare neurodevelopmental disorderPlan to initiate ...

A Phase 2 study evaluating TPST-1495 in patients with FAP is set to begin this year, conducted by the Cancer Prevention Clinical Trials Network and funded by the National Cancer Institute (NCI) ...

Scilex Holding said the Food and Drug Administration granted orphan drug designation for colchicine for the treatment of pericarditis. The company's Gloperba, licensed from Romeg Therapeutics, is a ...

Arkansas Gov. Sarah Huckabee Sanders has signed into law a piece of legislation that bars pharmacy benefit managers operating ...

The first patient with relapsed or refractory CD5-positive T-cell lymphoma has been dosed with the CAR-T cell therapy MB-105.

REC-4881 is an orally bioavailable, non-ATP-competitive allosteric small molecule MEK 1/2 inhibitor. The platform analyzed cellular models of APC gene loss-the root cause of FAP-to identify compounds ...

Rare diseases, often referred to as orphan diseases, affect small patient populations, yet collectively they impact more than ...

Under the Orphan Drug Act, the FDA may grant orphan designation to a drug or biological product intended to treat a rare disease or condition, which is generally a disease or condition that ...

Scilex Holding said the Food and Drug Administration granted orphan drug designation for colchicine for the treatment of pericarditis. The company's Gloperba, licensed from Romeg Therapeutics ...