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

BioMarin eyes 2019 FDA filing for haemophilia A gene therapy

BioMarin eyes 2019 FDA filing for haemophilia A gene therapy

During the update, BioMarin also laid out its expectations for vosoritide, a drug for short stature in children with achondroplasia in a phase 3 trial due to read out before the ... Achondroplasia is the leading cause of short stature, and is caused by

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  • BioMarin drug boosts growth in children with dwarfism BioMarin drug boosts growth in children with dwarfism

    The small study in children with achondroplasia - the most common form of dwarfism - showed that children treated with the highest dose of BMN 111 (vosoritide) grew 6cm in six months, while ... The results suggest vosoritide could a new treatment option

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