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PP26-3 : Efficacy and Safety of Asfotase Alfa in Patients with Infantile Hypophosphatasia Treated for up to 3.5 Years: Results from a Phase II, Open-Label, Uncontrolled Study

Researchers

Presenter

  • Christine Hofmann

Principal Investigators

  • Paul Harmatz

  • Gerard Vockley

  • Hui Zhang

  • Kenji Fujita

  • Cheryl Rockman-Greenberg

Medical Centers

  • Childrens Hospital, University of Wuerzburg, Wuerzburg, Germany

  • Children's Hosp of Pittsburgh of UPMC, Pittsburgh, PA

  • UCSF Benioff Childrens Hospital Oakland, Oakland, CA

Locations

  • United States

  • Canada

  • Germany

Companies

  • Alexion Pharmaceutic

Study Components

Therapeutic Area

  • Genetic Disorder

  • Musculoskeletal

Disease

  • Hypophosphatasia infantile type

Biomarkers

  • N/A

Drug/Treatment

  • Alkaline phosphatase

  • Strensiq

Outcome

  • N/A

Study Design

  • open label

Phase

  • NA

Study Id's

  • N/A

Sponsors

  • N/A

Result

  • Interim

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