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Latest From Ipsen SA

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Rocket's all-stock acquisition of Renovacor is valued at about $53m and adds a preclinical portfolio targeting genetically driven cardiovascular disease.

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Focused on rare liver diseases such as acute-on-chronic liver failure following its 2020 disappointment in NASH, Genfit looks to become sector leader in ACLF and other indications such as hepatic encephalopathy.

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Cardiff Oncology Falls Despite Onvansertib’s Broad Potential In KRAS-Mutant CRC

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Overcoming Rare Disease Trial Recruitment Challenges

Rare disease treatments have been increasingly attractive targets for investment, but clinical trials of these agents are laden with unique challenges, not least of which is that there are by definition small and possibly disparately located patient populations. However, by cultivating relationships with patient organizations and carefully designing studies, companies in the rare disease space are proving they can overcome logistical hurdles. 

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Company Information

  • Industry
  • Biotechnology
  • Pharmaceuticals
  • Pharmaceuticals
    • OTC, Consumer
    • Specialty Pharmaceuticals
  • Other Names / Subsidiaries
    • Clementia Pharmaceuticals, Inc.
    • Epizyme, Inc.
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    • Sterix
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    • Syntaxin Ltd.
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    • Octagen Corporation
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