Patients in the US with ultra-rare blood clotting disorder congenital thrombotic thrombocytopenic purpura (cTTP) have an FDA-approved therapy for the first time, after Tak
The FDA has started a priority review of X4 Pharma’s mavorixafor, angling to become the first therapy for the rare disease WHIM syndrome, with a decision due by 30th April
Ipsen has said that it plans to file a new marketing application for its treatment for itch in Alagille syndrome (ALGS), Bylvay, after failing to persuade an EMA committee
Belgian biopharma UCB has been given the green light by the FDA for Zilbrysq as a treatment for generalised myasthenia gravis (gMG), one of two drugs that the company hope
Japan’s Kyowa Kirin has reached an agreement to buy Orchard Therapeutics of the UK in a deal valued at up to $478 million, ahead of an FDA decision on the biotech’s lead g
Ionis Pharma is preparing to file for regulatory approval of its experimental therapy olezarsen for familial chylomicronaemia syndrome (FCS) in the US and Europe after the
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