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Ultragenyx

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

Sanofi’s rare blood disorder drug Cablivi set for European approval

Sanofi’s rare blood disorder drug Cablivi set for European approval

Among the other recommendations, Ultragenyx picked up a positive CHMP opinion for its mucopolysaccharidosis type VII drug – Mepsevii (vestronidase alfa) – which was approved in the US last November.

Latest news

  • Rigel and Ultragenyx claim approvals for rare drugs Rigel and Ultragenyx claim approvals for rare drugs

    Rigel and Ultragenyx claim approvals for rare drugs. Tavalisse and Crysvita are set to treat patients with chronic ITP and XLH, respectively. ... The FDA gave the go-ahead to a pair of rare disease therapies this week, both marking the first-ever

  • Archive listing

    Rigel and Ultragenyx claim approvals for rare drugs.

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Latest Intelligence

  • Deal Watch October 2015 Deal Watch October 2015

    The research collaboration between Arcturus Therapeutics and Ultragenyx Pharmaceutical covers the discovery and development of mRNA therapeutics to certain rare disease targets using the Arcturus UNA Oligomer chemistry and LUNAR nanoparticle

  • Pharma deals during January 2013 Pharma deals during January 2013

    Deal Type. Comments. Baylor Research Institute. Ultragenyx. Collaboration. Triheptanoin UX 007 in rare and ultra-rare diseases. ... For example, Ultragenyx has in-licensed triheptanoin for long-chain fatty acid oxidation disorders from the Baylor

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Latest appointments

  • Genentech’s Sunil Agarwal joins rare disease firm Genentech’s Sunil Agarwal joins rare disease firm

    Genentech’ s Sunil Agarwal joins rare disease firm. He will serve as chief medical officer at Ultragenyx. ... Dr Sunil Agarwal has left his senior role at Genentech to join rare disease specialist Ultragenyx Pharmaceutical as its chief medical officer.

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