News Sentynl gets first FDA approval for rare disease Menkes Children with a devastating, rare disorder of copper metabolism, called Menkes disease, now have an FDA-approved treatment for the first time.
News Hutchmed scores with drug for rare autoimmune disease Hutchmed is preparing to file for approval of its drug for the rare disorder wAIHA in China, after chalking up a win in a pivotal phase 2/3 trial.
News FDA declines to approve Corcept's rare disorder drug As the celebrations for the end of 2025 gathered pace, the FDA delivered a year-end blow to Corcept by turning down its new Cushing's drug.
News Ultragenyx, Mereo's brittle bone drug flames out in trials Two phase 3 trials of Ultragenyx and Mereo's sclerostin inhibitor setrusumab in osteogenesis imperfecta missed their primary efficacy objectives.
News BioMarin ends year with $4.8bn play for Amicus BioMarin takes a leap towards its $4bn revenue target with a $4.8bn deal to acquire Amicus, which has two rare disease therapies on the market.
News Kyverna rises on stiff person syndrome data Kyverna hopes to file CAR-T therapy miv-cell for approval as the first therapy for stiff person syndrome, a rare and debilitating autoimmune disease.
Patients Bringing parity to infertility, with Jean Duvall During JPM2026, pharmaphorum spoke with Jean Duvall, CEO of Repronovo, a company that’s working on new infertility treatments for men and women.
Sales & Marketing Sponsored How to meet your physicians where they are online A pharma company’s core mission is to improve patient outcomes. This hinges on effectively influencing HCP clinical behaviour and driving disease education.