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Wilson's disease

This page shows the latest Wilson's disease news and features for those working in and with pharma, biotech and healthcare.

AZ’s new rare disease unit wins in Wilson disease

AZ’s new rare disease unit wins in Wilson disease

AstraZeneca’s new rare disease unit, formed through the $39bn purchase of Alexion Pharmaceuticals, had announced positive results from a phase 3 trial in Wilson disease. ... Wilson disease is a rare, progressive genetic condition in which the body’s

Latest news

  • Pfizer buys dwarfism drug company Therachon for $810m Pfizer buys dwarfism drug company Therachon for $810m

    The deal adds to Pfizer’s rare disease portfolio, which just chalked up a major success with the approval of tafamidis products Vyndaqel and Vyndamax for transthyretin amyloid cardiomyopathy (ATTR-CM) ... TA-46 will add to other pipeline candidates

  • Alexion adds to pipeline with $1.2bn Syntimmune buy Alexion adds to pipeline with $1.2bn Syntimmune buy

    Alexion used a priority review voucher from an earlier rare disease therapy approval to accelerate the FDA’s deliberations and bring forward a possible approval date. ... $855m, adding orally-active copper binder WTX-101 in phase 3 testing for genetic

  • Alexion adds to pipeline rebuild with $855m Wilson buy Alexion adds to pipeline rebuild with $855m Wilson buy

    Also acquires the Swedish group’s lead product for Wilson’s disease. Alexion Pharma’s drive to reduce its reliance on cash cow Soliris has gone up a gear with a ... Sweden-based Wilson’s lead product is WTX101, currently in phase III development

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