Rare diseases have become an attractive target for pharmaceutical companies both in Australia and internationally with ...

Its architect's daughter has cystic fibrosis—and benefits from a "miracle drug" backed by an agency he's attacking.

KALA BIO, Inc. (NASDAQ:KALA), a clinical-stage biopharmaceutical company dedicated to the research, development and ...

We think Biogen's neurology, immunology, and focus on rare diseases support a narrow moat. Biogen's strategy has its roots in the 2003 merger of Biogen (Avonex for treating multiple sclerosis, or MS) ...

One in 10 Americans live with a rare disease, which often lacks effective treatments. Such is the case for PIGA-CDG, an ultra ...

FY 2024 total sales growth of 9.9% at CER1, or 8.7% as reported, with growth driven by strong performance across all therapeutic areas, including ...

Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or ...

Calvin Sandra, the Senior Vice President and Chief Accounting Officer at Travere Therapeutics, Inc. (NASDAQ:TVTX), executed a series of transactions involving the company's common stock. The ...

Accumulation of fat molecules is detrimental to the cell. Researchers have made a breakthrough in understanding how our cells manage to stay healthy by recycling important fat molecules. Their study ...

Biomedical engineers at Duke University have demonstrated a promising new approach that could be used to treat a rare and complex class of genetic ...