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AEP1019 : A global natural history study of Fibrodysplasia Ossificans Progressiva (FOP): 12-month outcomes

Researchers

Presenter

  • Mona Al Mukaddam

Principal Investigators

  • Robert J. Pignolo

  • Genevive Baujat

  • Matthew A Brown

  • Carmen De Cunto

  • Maja Di Rocco

  • Edward C. Hsiao

  • Richard Keen

  • Kim-Hanh Le Quan Sang

  • Andrew Strahs

  • Rose Marino

  • Frederick S. Kaplan

Medical Centers

  • Departments of Orthopaedic Surgery and Genetics, Center for Research in FOP and Related Disorders, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA

  • Department of Biomedical Statistics, Mayo Clinic, Rochester, Minnesota, United States

  • Département de Génétique, Institut IMAGINE and Hôpital Universitaire Necker-Enfants Malades, Paris, France

  • Guy’s & St Thomas’ NHS Foundation Trust and King’s College London NIHR Biomedical Research Centre, London, United Kingdom

  • Pediatric Rheumatology Section, Department of Pediatrics, Hospital Italiano de Buenos Aires, Buenos Aires, Argentina

  • Unit of Rare Diseases, Department of Pediatrics, Giannina Gaslini Institute, Genoa, Italy

  • Division of Endocrinology and Metabolism, UCSF Metabolic Bone Clinic, Institute of Human Genetics, and UCSF Program in Craniofacial Biology, Department of Medicine, University of California-San Francisco, San Francisco, CA, United States

  • Centre for Metabolic Bone Disease, Royal National Orthopaedic Hospital, Stanmore, United Kingdom

  • Ipsen, Boston, MA, United States

Locations

  • United States

  • France

  • United Kingdom

  • Argentina

  • Italy

Companies

  • N/A

Study Components

Therapeutic Area

  • Musculoskeletal

Disease

  • Fibrodysplasia ossificans progressiva

Biomarkers

  • N/A

Drug/Treatment

  • N/A

Outcome

  • N/A

Study Design

  • Double Blind/Blinded

Phase

  • NA

Study Id's

  • N/A

Sponsors

  • N/A

Result

  • N/A

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