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achondroplasia

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

Pfizer buys dwarfism drug company Therachon for $810m

Pfizer buys dwarfism drug company Therachon for $810m

trap that acts as a decoy to dampen down signals that suppress bone growth in people with achondroplasia. ... There are currently no approved treatments for achondroplasia, which affects an estimated 250, 000 people worldwide, according to Pfizer.

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  • 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

  • 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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Webinar: Usability and UX: You’ve been asked to test...What next?
Thursday 27th June 2019 10am EDT / 3pm BST / 4pm CEST...
Event: EphMRA Annual Conference
We're sponsoring, exhibiting and presenting three papers at this year’s EphMRA Annual Conference in Warsaw, Poland....
Research Partnership and Janssen awarded runner-up in MR Excellence Awards
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