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 ...

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

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

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

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) ...

FDA Orphan Drug Designation status is granted for treatments of rare diseases affecting fewer than 200,000 people in the U.S. This designation provides NS Pharma with a seven-year market ...

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 ...

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 ...