BioMarin Pharmaceutical has struck a deal to buy fellow biotechnology company Amicus Therapeutics for about $4.8 billion in ...
It’s the second acquisition for BioMarin in 2025, as the company seeks to diversify its product portfolio after the ...
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 ...
Ipsen’s oral small molecule has failed to reduce the progression of a genetic disease that gradually turns tendons and ...
Gene editing has the potential to cure thousands of people suffering from devastating rare diseases. But the Nobel ...
This year, gene-editing technology was customized to fix mutations in a single patient’s genes for the first time.
San Rafael-based BioMarin is putting down a hefty all-cash wager on the rare-disease market, agreeing to buy New Jersey ...
Lozano is a rare disease mom, neuroscience Ph.D. candidate at UC Davis, and board member for the PURA Syndrome Foundation. In May, a historic moment in science and medicine was captured in a single ...
Backed by a handful of prominent investors including the Gates Foundation, the startup is pursuing an approach it believes ...
Rare neurological diseases offer unusually clear models for testing earlier diagnosis, precision treatment and long-term ...
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.
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