Rare diseases

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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.

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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.

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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.

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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.

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FDA clears first gene therapy for rare genetic disease WAS

Fondazione Telethon's Waskyra has become the first gene therapy in the US for Wiskott-Aldrich syndrome, and the first taken to market by a non-profit.

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Bayer takes Alport syndrome drug into phase 2

Bayer has started a phase 2a trial of an antibody that it hopes could provide a targeted, potentially disease-modifying therapy for Alport syndrome.

FDA declines to approve Corcept's rare disorder drug

Ultragenyx, Mereo's brittle bone drug flames out in trials

BioMarin ends year with $4.8bn play for Amicus

Kyverna rises on stiff person syndrome data

FDA clears first gene therapy for rare genetic disease WAS

Bayer takes Alport syndrome drug into phase 2

FDA points to liver injury with Amgen's Tavneos

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Rare diseases

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