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

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

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.

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

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

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

Pharmed Foundation and ORD India launched the KK Aurora Light of Hope Fund. The CSR programme will provide free or subsidised ...

There are long-running TV shows, and then there's Grey's Anatomy. The ABC medical drama is one of the longest-running TV shows of all time and during the run Grey's has had wild medical cases, ...

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

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, ...

Past the red carpets and the flashing lights of the paparazzi, some celebrities live in a reality more complicated than what is shown. A number of them live with medical conditions only seen in a tiny ...

When you buy through links on our articles, Future and its syndication partners may earn a commission. During the early episodes of Grey's Season 2, much of the drama was surrounding Addison's arrival ...