Measuring outcomes in ultra-rare bone diseases: Methodology of the palovarotene fibrodysplasia ossificans progressiva clinical development programme

• Robert J. Pignolo,
• Geneviève Baujat,
• Matthew A. Brown,
• Carmen De Cunto,
• Maja Di Rocco,
• Edward C. Hsiao,
• Richard Keen,
• Mona Al Mukaddam,
• Andrew Strahs,
• Donna R. Grogan,
• Rose Marino,
• Frederick S. Kaplan

Affiliations

Robert J. Pignolo

Department of Medicine, Mayo Clinic, Rochester, United States

Geneviève Baujat

Departement de Genetique, Institut IMAGINE and Hôpital Necker-Enfants Malades, Paris, France; Corresponding author.

Matthew A. Brown

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

Carmen De Cunto

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

Maja Di Rocco

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

Edward C. Hsiao

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

Richard Keen

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

Mona Al Mukaddam

Departments of Orthopaedic Surgery & Medicine, The Center for Research in FOP and Related Disorders, Perelman School of Medicine, University of Pennsylvania, Philadelphia, United States

Andrew Strahs

Clementia Pharmaceuticals Inc., Newton, United States

Donna R. Grogan

Clementia Pharmaceuticals Inc., Newton, United States

Rose Marino

Clementia Pharmaceuticals Inc., Newton, United States

Frederick S. Kaplan

Departments of Orthopaedic Surgery & Medicine, The Center for Research in FOP and Related Disorders, Perelman School of Medicine, University of Pennsylvania, Philadelphia, United States