A new study challenges what's long been assumed about genetic variants thought to always cause inherited blindness.

Breakthrough research is bringing new hope this Christmas to a Grand Blanc family facing a rare neurological disease.

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

Combined, the two drugs made revenues of nearly $600 million in the last four quarters, while BioMarin has forecast that its ...

A powerful profile of Dr. David Fajgenbaum using AI drug repurposing to cure rare diseases and transform medicine.

As the Q3 earnings season comes to a close, it’s time to take stock of this quarter’s best and worst performers in the ...

Discover why Mirum Pharmaceuticals, Inc. stock surged 65%. Explore robust pipeline growth, revenue outlook, and analyst ...

Amgen Inc ($AMGN) announced an update on their ongoing clinical study. Amgen’s latest clinical move targets a niche but ...

Just two months after reviving its prowess in the idiopathic pulmonary fibrosis (IPF) treatment area with rare lung disease ...

However, significant changes have been underway, converging regulatory breakthroughs, maturing AI capabilities, and a broader reconsideration of how therapies are developed and who they serve.

Policy initiatives have come fast and furious at the FDA this year. While guidances on rare diseases and vaccines have consumed most of the ink, policy shifts aimed at improving FDA efficiencies and ...

BioMarin Pharmaceutical’s strong rare disease drug portfolio and Amicus acquisition drive a ‘Buy’ rating. Click here to read my latest analysis of BMRN stock.