News EU kicks off review of GSK's linerixibat in PBC GSK's drug for severe itching in patients with autoimmune liver disease PBC is now under review in the EU and US, as Mirum Pharma tries to catch up.
News GSK reveals data behind linerixibat for PBC 'relentless itch... GSK reveals the data behind its linerixibat for itching associated with autoimmune liver disease PBC as it anticipates first approvals for the drug.
News FDA clears first therapy for rare disease CTX from Mirum Mirum Pharma's Ctexli has become the first FDA-approved therapy for ultra-rare disease cerebrotendinous xanthomatosis (CTX).
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.
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