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Overcoming barriers to conducting clinical trials in childhood rare disease research
demonstrating the effectiveness of a medication in reducing disease progression and liver transplants in children with Alagille syndrome (ALGS). "By using a global database, we were able to mimic ...
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Ipsen crosses EU finish line with Alagille syndrome drug
in patients with the rare liver disease Alagille syndrome (ALGS). Kayfanda (odevixibat) is labelled in the EU to treat cholestatic pruritus – itching caused by impaired bile formation or flow ...