Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease that affects red blood cells, causing symptoms like dark urine, ...

When a test determined that Walter had hemophilia, his Omaha owner joined a group seeking to raise $50,000 for a clinical ...

Adcendo, a biotech company pioneering the development of first and best-in-class ADCs for cancers with a high unmet medical ...

The Food and Drug Administration (FDA) has granted Orphan Drug designation to EXG-34217 for the treatment of telomere biology disorders.

Pfizer opted to discontinue the product for "several reasons," including limited interest from patients and physicians in gene therapies for hemophilia.