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Ultragenyx Pharmaceutical Inc.

http://www.ultragenyx.com/

Latest From Ultragenyx Pharmaceutical Inc.

Push To Create Ultra-Rare Orphan Drug Designation Finds Resistance At US FDA

Agency officials don’t think a new definition or incentives will fix the issues related to development in rare diseases with only a handful of patients, as advocates continue pushing the idea.

Rare Diseases Review Pathway

A Passion For Patient Care And De-Risking Disruptive Technologies

Biomedical engineering graduate Cécile Dupont has for the past 12 years helped healthcare technology businesses develop disruptive solutions that positively impact patients’ lives. The current chapter of her career opened in 2017 when she joined Sofinnova MD Start, the early-stage medtech incubator of VC investor Sofinnova Partners, where she is a partner.

Rising Leaders Innovation

Ultragenyx Faces Henrietta Lacks Suit Modeled On Action Against Thermo Fisher

While the unjust enrichment claim against Ultragenyx uses the same argument the estate of Henrietta Lacks employed against Thermo Fisher, resulting in a settlement earlier this month, one big difference between the cases is that Ultragenyx does not yet have a commercial product that relies on the HeLa cells.

Legal Issues Intellectual Property

US FDA External Controls Guidance Does Little To Advance The Cause, Especially In Rare Diseases – Comments

Industry and patient groups take issue with draft guidance’s skeptical tone on utility and reliability of externally controlled studies, with some saying the guidance ignores special considerations inherent in studying rare diseases. Recommendations on access to patient-level data for external control arms also are problematic and likely infeasible, stakeholders say.

Real-World Evidence Rare Diseases
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Company Information

  • Industry
  • Biotechnology
  • Pharmaceuticals
  • Biotechnology
    • Gene Therapy, Cell Therapy
    • Large Molecule
      • Antibodies
  • Other Names / Subsidiaries
    • Dimension Therapeutics
    • GeneTx Biotherapeutics LLC
    • Ultragenyx
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