AstraZeneca said on Aug. 26 a late-stage trial showed the company’s medicine for a rare disease that causes accumulation of copper in the body was three times more successful in helping remove deposits from tissues compared with standard care.

International biopharmaceutical firm Genevant Sciences announced a global collaboration and licensing deal with Takeda Pharmaceutical to create and commercialize new therapies that would hopefully treat two undisclosed types of rare liver diseases.

Merck won U.S. Food and Drug Administration approval for a first-of-its-kind oral hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor that is used to treat a rare genetic disease that causes benign and cancerous tumors in blood vessels associated with renal cell carcinoma.

After discussions with the U.S. Food and Drug Administration, Paris-based Ipsen withdrew the company’s New Drug Application (NDA) for palovarotene.

BioCryst Pharmaceuticals, a global biopharmaceutical company that focuses on developing treatments for rare diseases, canceled plans to hold a public offering to raise funds. 

The Greater Than One Group and its non-profit subsidiary Honeycomb Health will be launching a cross country road trip to raise money for the rare disease cause.

Europe’s medicines regulator added a rare nerve-degenerating disorder, Guillain-Barré syndrome, as a possible rare side effect from Johnson & Johnson’s Covid-19 vaccine after the European Medicines Agency reviewed 108 cases reported worldwide.

Boston-based Alexion Pharmaceuticals announced positive topline data from the company’s Phase III trial of Ultomiris (ravulizumab-cwvz) in adults with generalized myasthenia gravis.

A study found potential in a novel gene therapy method to help children born with a rare genetic and neurodegenerative disorder, AADC deficiency.

The U.S. Food and Drug Administration is planning a new warning for Johnson & Johnson’s Covid-19 vaccine over a rare but serious side effect, Guillain-Barré syndrome (GBS).