Hemispherian AS, a pioneering biotech company developing next-generation therapeutics for aggressive cancers, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug ...

Paris: Sanofi has received orphan drug designation from the Ministry of Health, Labour and Welfare (MHLW) in Japan for ...

The Senate Parliamentarian reversed course on a small provision in the One Big Beautiful Bill Act that could boost cures for rare diseases.

The Railroad Commission’s program for sealing orphan oil and gas wells will get an additional $100 million over the next two ...

As recently announced, initial proof-of-concept results from this ongoing investigator-initiated trial (IIT) show crofelemer ...

My story began at Baby House No. 1 in Novosibirsk, Russia, one of thousands of children left behind in the wake of the Soviet ...

My story began at Baby House No. 1 in Novosibirsk, Russia, one of thousands of children left behind in the wake of the Soviet ...

Emapalumab is approved for both adults and pediatric (newborn and older) patients with MAS in Still disease with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS.

The FDA has approved Gamifant as the first-ever treatment for macrophage activation syndrome in Still’s disease for adults ...

Gamifant marks the first-ever FDA-approved treatment for both adult and pediatric patients with hemophagocytic ...

Scientists recently released research showing where groundwater is most at risk of contamination by orphan wells.

The Orphan Cures Act has been added back into the Senate version of President Donald Trump’s “big, beautiful bill” after being ...