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

U.S. health officials have added two deadly rare diseases to the list of conditions the government recommends states screen ...

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.

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

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

The FDA has granted approval to a new gene therapy for a rare immune disorder, the maker of which is notably not a drug ...

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.

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 ...

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

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, ...

The seven-year medical odyssey that once defined rare disease diagnosis is being rewritten by algorithms capable of identifying medical zebras in the time it takes to refresh your social media feed.