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

This weird thing keeps happening where it feels like the sock in my left shoe has gotten wadded up underneath the ball of my foot. Every time I ...

We recently compiled a list of the 7 Most Promising Gene Editing Stocks According to Analysts. BioMarin Pharmaceuticals Inc.

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

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 ...

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.

A mom saved her two-year-old daughter’s life after she donated her liver. Elle Daniel, 35, gave her daughter Ruby a piece of her liver in June 2025, after the toddler was diagnosed with liver 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.

Now, Musunuru and colleagues are preparing a master clinical protocol to study a CRISPR therapy that's mostly the same as the one they used to treat baby KJ, except they plan to swap out the gene ...

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

Pakistan: Researchers from Pakistan have reported a rare and severe presentation of erosive lichen planus involving the eyes, ...