CDER Director Patrizia Cavazzoni and CBER Director Peter Marks provided an update on the rare disease innovation hub in a new FDA Voices blog post. FDA’s Patrizia Cavazzoni, director, Center for Drug ...

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It’s been over a dozen years since Kate Bush last released a new album, but as she revealed in a rare interview Friday, the Rock Hall-inducted singer may finally be readying her return to music.

Monopar Therapeutics is recovering a drug from the scrap heap of AstraZeneca’s rare disease pipeline. It has licensed ALXN-1840, a candidate for the treatment of Wilson disease that was ...

About Wilson Disease Wilson disease is a rare and progressive genetic condition in which the body’s pathway for removing excess copper is compromised. 1 It affects one in 30,000 live births in ...

AstraZeneca Rare Disease for an exclusive worldwide license to ALXN-1840 (bis-choline tetrathiomolybdate), a drug candidate for Wilson disease that Alexion has progressed through a Phase 3 ...

She said the protocol her team developed for Charlotte had dramatically improved the speed of diagnosing rare diseases to take it from five to 20 years, down to an average of less than 12 months.

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