Rare diseases

News

The SARCO41 study suggests Lilly's Verzenio could become the first targeted therapy for rare, aggressive tumour dedifferentiated liposarcoma.

News

Trials of AstraZeneca's anselamimab and Regeneron's Lynozyfic could deliver much-needed new therapeutic options for rare disease AL amyloidosis.

News

After a phase 2 readout, Sanofi is charting a route to market for AATD therapy efdoralprin alfa, acquired as part of its $2.2bn takeover of Inhibrx.

News

AstraZeneca may struggle to differentiate eneboparatide, a hypoparathyroidism drug from its $800m takeover of Amolyt, from Ascendis' rival Yorvipath.

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Partner's Bizengri has been approved for a rare form of bile duct cancer, two days after being awarded a national priority review voucher.

News

Patients in the EU with the ultra-rare disease WHIM syndrome now have their first approved treatment, Norgine's Xolremdi.