Randolph County hosted its annual “Hooked on Fishing Not on Drugs" event event on Saturday, offering an introduction to some outdoor activities for kids.

Govorestat is a central nervous system (CNS) penetrant Aldose Reductase Inhibitor (ARI) being developed for the treatment of multiple rare diseases including Charcot-Marie-Tooth Sorbitol Dehydrogenase ...

An EU committee is recommending that tranilast, the active ingredient in a pulmonary fibrosis drug candidate, be awarded ...

Treatment with the Avmapki Fakzynja Co-pack received accelerated FDA approval for previously treated low-grade serous ovarian ...

Hikma Pharmaceuticals has agreed to pay $50 million to settle a class action accusing the drugmaker of scheming to delay a ...

Dr. Gomez brings extensive neuroscience and orphan disease development experience to Raya as it plans multiple clinical trials in 2026. MONTREAL, QUEBEC, CANADA, May 8, 2025 /EINPresswire.com/ -- Raya ...

The FDA has granted Orphan Drug designation to BA-102 for the treatment of Phelan-McDermid syndrome, a rare neurodevelopmental disorder.

(TSE 4552, JCR) today announced that the U.S. Food and Drug Administration (FDA) granted orphan drug designation (ODD) to JR-446, an investigational drug for the treatment of mucopolysaccharidosis ...

The FDA has granted Orphan Drug designation to TPST-1495, dual receptor inhibitor of prostaglandin signaling, for the treatment of familial adenomatous polyposis.

Treatment with givinostat has received fast track designation from the United States Food and Drug Administration for ...

FDA Commissioner Marty Makary’s proposal to roll out a new drug approval pathway is heightening hope for the rare disease ...