2026 is shaping up to be a pivotal year for rare disease drugmakers, with key approvals, filings and readouts lined up for ...

Full pipelines and rising revenues lead rare disease biotechs to restyle themselves as more mature, global biopharma ...

When Jesy Nelson shared an emotional Instagram video in January 2026, the former Little Mix singer did more than reveal a ...

Lozano is a rare disease mom, neuroscience Ph.D. candidate at UC Davis, and board member for the PURA Syndrome Foundation. In May, a historic moment in science and medicine was captured in a single ...

Danish dermatology specialist Leo Pharma and its CEO Christophe Bourdon came to San Francisco to attend the J.P. Morgan ...

By identifying the microbes and metabolic pathways responsible for a rare, gut-induced condition known as auto-brewery ...

GeneDx, which specializes in diagnostics for rare and ultra-rare genetic disorders, is helping to get kids the diagnostic attention they need as part of Diagnosis is Power. Currently, one in 10 ...

No standard treatments for the sometimes deadly disorder have been developed, but that may change due to the newly published ...

Torie Bosch is the First Opinion editor at STAT. Celena Lozano’s son Benny, who turned 5 in November, loves trains, trucks — anything that goes. He also has a rare disease, PURA syndrome. Earlier this ...

Rare diseases are defined as conditions affecting fewer than 200,000 people in the US or less than 1 in 2,000 in Europe. 1-2 While each disease is individually rare, collectively, they represent a ...

Corcept Therapeutics (NASDAQ: CORT) and Amicus Therapeutics (NASDAQ: FOLD) delivered Q3 2025 results highlighting two rare ...