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

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.

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

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 ...

Ozempic® can cause gallbladder diseases, including gallstones and gallbladder inflammation. Gallbladder problems are rare — 0 ...

The FDA is expected to decide on treatments for breast cancer, EBV+ post-transplant lymphoproliferative disease, focal segmental glomerulosclerosis, presbyopia, and severe allergic reactions.

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

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