Results show that targeting the underlying cause of Dravet syndrome with zorevunersen may improve outcomes for people with this rare, ...

U.S. FDA places FREEDOM2 trial on partial clinical hold related to preclinical pharmacology and toxicology -- Received regulatory clearance to initiate FREEDOM2 in South Korea, Australia, and New ...

Namodenoson is a highly selective A3 adenosine receptor (A3AR) agonist, which has shown a compelling safety profile and demonstrated anti-tumor activity in preclinical pancreatic cancer models. The ...

Rare Disease Day, observed annually on the last day of February, serves as an international campaign to raise awareness about the challenges faced by individuals and families living with rare diseases ...

Discover how falling cocoa prices, sustainability shifts, and innovation in cocoa alternatives are reshaping the future of ...

A Senate hearing probed whether the Food and Drug Administration's process for approving rare-disease therapies is ...

Hint: The products themselves may not be the issue.

Andy Dick said his heart stopped during an apparent overdose in December, leading him to be diagnosed with "five to seven ...

(Nasdaq: EYPT), a company committed to developing and commercializing innovative therapeutics to improve the lives of patients with serious retinal diseases, today announced the first patient dosed in ...

Hansa Biopharma AB, ("Hansa" or "the Company"), (Nasdaq Stockholm: HNSA), today announced that the Food and Drug Administration (FDA) ...

You’ve probably seen a headline or two about an invasive species taking over. Perhaps you’ve seen a sign warning of ...