The Amicus acquisition will bolster BioMarin’s rare disease portfolio, while potentially supporting the company’s revenue ...

As the FDA unveils a parade of initiatives aimed at accelerating drug development for rare diseases, experts appeal for a consistent approval process that will support and further catalyze momentum.

BioMarin is acquiring Amicus Therapeutics for $4.8 billion, merging two rare disease-focused biotechs founded around the turn ...

Ipsen’s oral small molecule has failed to reduce the progression of a genetic disease that gradually turns tendons and ...

BioMarin Pharmaceutical said on Friday it would acquire Amicus Therapeutics for about $4.8 billion, in the drugmaker's second deal this year to expand its presence in rare diseases.

Gene editing has the potential to cure thousands of people suffering from devastating rare diseases. But the Nobel ...

Backed by a handful of prominent investors including the Gates Foundation, the startup is pursuing an approach it believes ...

Cure Rare Disease (CRD) announced it has been awarded a $7.4 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the development of a novel gene therapy for ...

Rare neurological diseases offer unusually clear models for testing earlier diagnosis, precision treatment and long-term ...

San Rafael-based BioMarin is putting down a hefty all-cash wager on the rare-disease market, agreeing to buy New Jersey ...

Soleo Health has appointed Jody Thompson as senior vice president of business development, rare disease, to broaden the company’s portfolio of exclusive and limited distribution therapies for rare, ...

MS. JOHNSON: Hello, and welcome to Post Live. I’m Akilah Johnson, a national health reporter focused on health disparities here at The Washington Post. And today we have two segments on rare diseases.