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beta-thalassemia

This page shows the latest beta-thalassemia news and features for those working in and with pharma, biotech and healthcare.

Orchard plans IPO as gene therapy pipeline advances

Orchard plans IPO as gene therapy pipeline advances

beta thalassaemia – which has just bagged a Priority Medicines (PRIME) designation from the EMA. ... It has been tested so far in nine patients with transfusion-dependent beta-thalassemia, the most severe form of the disease.

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  • Bluebird Bio: on the cusp of a gene therapy revolution Bluebird Bio: on the cusp of a gene therapy revolution

    In 2015 an advisory panel recommended the delay of trials in children with beta thalassemia for 1-2 years until more safety data could be obtained. ... Raju Prasad says Bluebird can offer a compelling cost-effectiveness case for its treatment in beta

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