Long-term use of interleukin-1 inhibitors reduce flare activity in patients with fibrodysplasia ossificans progressiva.

Autor: Haviv R; Pediatric Rheumatology Unit, Department of Pediatrics, Meir Medical Center, Kfar Saba, Israel.; School of Medicine, Faculty of Medical and Health Sciences, Tel Aviv University, Tel Aviv, Israel., Zeitlin L; School of Medicine, Faculty of Medical and Health Sciences, Tel Aviv University, Tel Aviv, Israel.; Pediatric Orthopedic Department, Dana-Dwek Children's Hospital, Sourasky Medical Center, Tel Aviv, Israel., Moshe V; Pediatric Rheumatology Unit, Department of Pediatrics, Meir Medical Center, Kfar Saba, Israel., Ziv A; Pediatric Rheumatology Unit, Department of Pediatrics, Meir Medical Center, Kfar Saba, Israel.; School of Medicine, Faculty of Medical and Health Sciences, Tel Aviv University, Tel Aviv, Israel., Rabinowicz N; Pediatric Rheumatology Unit, Department of Pediatrics, Meir Medical Center, Kfar Saba, Israel., De Benedetti F; Division of Rheumatology, Bambino Gesù Children's Hospital IRCCS, Rome, Italy., Prencipe G; Division of Rheumatology, Bambino Gesù Children's Hospital IRCCS, Rome, Italy., Matteo V; Division of Rheumatology, Bambino Gesù Children's Hospital IRCCS, Rome, Italy., De Cunto CL; Rheumatology Section, Department of Pediatrics, Hospital Italiano de Buenos Aires, Buenos Aires, Argentina., Hsiao EC; Division of Endocrinology and Metabolism, Institute for Human Genetics, The Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research, Department of Medicine, University of California, San Francisco, USA., Uziel Y; Pediatric Rheumatology Unit, Department of Pediatrics, Meir Medical Center, Kfar Saba, Israel.; School of Medicine, Faculty of Medical and Health Sciences, Tel Aviv University, Tel Aviv, Israel.

Publikováno v: Rheumatology (Oxford, England) [Rheumatology (Oxford)] 2024 Sep 01; Vol. 63 (9), pp. 2597-2604.

Most Fractures Treated Nonoperatively in Individuals With Fibrodysplasia Ossificans Progressiva Heal With a Paucity of Flareups, Heterotopic Ossification, and Loss of Mobility.

Autor: Lindborg CM; Department of Orthopaedic Surgery, Perelman School of Medicine, the University of Pennsylvania, Philadelphia, PA, USA.; The Center for Research in FOP and Related Disorders, Perelman School of Medicine, the University of Pennsylvania, Philadelphia, PA, USA., Al Mukaddam M; Department of Orthopaedic Surgery, Perelman School of Medicine, the University of Pennsylvania, Philadelphia, PA, USA.; The Center for Research in FOP and Related Disorders, Perelman School of Medicine, the University of Pennsylvania, Philadelphia, PA, USA.; Department of Medicine, Perelman School of Medicine, the University of Pennsylvania, Philadelphia, PA, USA.; Département de Genetique, Institut IMAGINE and Hôpital Universitaire Necker-Enfants Malades, Paris, France.; Division of Pediatric Orthopaedics, Seoul National University Children's Hospital, Seoul, Republic of Korea.; Department of Pediatrics, Pediatric Rheumatology Section, Hospital Italiano de Buenos Aires, Buenos Aires, Argentina.; Hospital Israelita Albert Einstein, Instituto de Ensino e Pesquisa, São Paulo, Brazil.; Department of Internal Medicine, Division of Endocrinology, Amsterdam University Medical Center, VU University Medical Center, Amsterdam, the Netherlands.; Rehabilitation Services Bureau, National Rehabilitation Center for Persons with Disabilities, Tokorozawa City, Japan.; Division of Endocrinology, Diabetes, and Metabolism, the Institute for Human Genetics, the Ely and Edythe Broad Institute for Regeneration Medicine, the Program in Craniofacial Biology and the Department of Medicine, University of California, San Francisco, CA, USA.; Department of Pediatrics, Klinikum Garmisch-Partenkirchen, Garmisch-Partenkirchen, Germany.; Department of Orthopaedic Surgery, the University of Cape Town, Cape Town, South Africa and Department of Rheumatology, Red Cross Children's Hospital, Cape Town, South Africa.; Institute for Experimental Endocrinology, Charite University Hospital, Berlin, Germany.; Department of Pediatrics, Institute of Medical Sciences, Medical College of Rzeszow University, University of Rzeszow, Rzeszow, Poland.; Department of Human Immunology, Institute of Medical Sciences, Medical College of Rzeszow University, University of Rzeszow, Rzeszow, Poland.; Department of Medicine, Mayo Clinic College of Medicine, Mayo Clinic, Rochester, MN, USA., Baujat G; Département de Genetique, Institut IMAGINE and Hôpital Universitaire Necker-Enfants Malades, Paris, France., Cho TJ; Division of Pediatric Orthopaedics, Seoul National University Children's Hospital, Seoul, Republic of Korea., De Cunto CL; Department of Pediatrics, Pediatric Rheumatology Section, Hospital Italiano de Buenos Aires, Buenos Aires, Argentina., Delai PLR; Hospital Israelita Albert Einstein, Instituto de Ensino e Pesquisa, São Paulo, Brazil., Eekhoff EMW; Department of Internal Medicine, Division of Endocrinology, Amsterdam University Medical Center, VU University Medical Center, Amsterdam, the Netherlands., Haga N; Rehabilitation Services Bureau, National Rehabilitation Center for Persons with Disabilities, Tokorozawa City, Japan., Hsiao EC; Division of Endocrinology, Diabetes, and Metabolism, the Institute for Human Genetics, the Ely and Edythe Broad Institute for Regeneration Medicine, the Program in Craniofacial Biology and the Department of Medicine, University of California, San Francisco, CA, USA., Morhart R; Department of Pediatrics, Klinikum Garmisch-Partenkirchen, Garmisch-Partenkirchen, Germany., de Ruiter R; Department of Internal Medicine, Division of Endocrinology, Amsterdam University Medical Center, VU University Medical Center, Amsterdam, the Netherlands., Scott C; Department of Orthopaedic Surgery, the University of Cape Town, Cape Town, South Africa and Department of Rheumatology, Red Cross Children's Hospital, Cape Town, South Africa., Seemann P; Institute for Experimental Endocrinology, Charite University Hospital, Berlin, Germany., Szczepanek M; Department of Pediatrics, Institute of Medical Sciences, Medical College of Rzeszow University, University of Rzeszow, Rzeszow, Poland., Tabarkiewicz J; Department of Human Immunology, Institute of Medical Sciences, Medical College of Rzeszow University, University of Rzeszow, Rzeszow, Poland., Pignolo RJ; Department of Medicine, Mayo Clinic College of Medicine, Mayo Clinic, Rochester, MN, USA., Kaplan FS; Department of Orthopaedic Surgery, Perelman School of Medicine, the University of Pennsylvania, Philadelphia, PA, USA.; The Center for Research in FOP and Related Disorders, Perelman School of Medicine, the University of Pennsylvania, Philadelphia, PA, USA.; Department of Medicine, Perelman School of Medicine, the University of Pennsylvania, Philadelphia, PA, USA.; Département de Genetique, Institut IMAGINE and Hôpital Universitaire Necker-Enfants Malades, Paris, France.; Division of Pediatric Orthopaedics, Seoul National University Children's Hospital, Seoul, Republic of Korea.; Department of Pediatrics, Pediatric Rheumatology Section, Hospital Italiano de Buenos Aires, Buenos Aires, Argentina.; Hospital Israelita Albert Einstein, Instituto de Ensino e Pesquisa, São Paulo, Brazil.; Department of Internal Medicine, Division of Endocrinology, Amsterdam University Medical Center, VU University Medical Center, Amsterdam, the Netherlands.; Rehabilitation Services Bureau, National Rehabilitation Center for Persons with Disabilities, Tokorozawa City, Japan.; Division of Endocrinology, Diabetes, and Metabolism, the Institute for Human Genetics, the Ely and Edythe Broad Institute for Regeneration Medicine, the Program in Craniofacial Biology and the Department of Medicine, University of California, San Francisco, CA, USA.; Department of Pediatrics, Klinikum Garmisch-Partenkirchen, Garmisch-Partenkirchen, Germany.; Department of Orthopaedic Surgery, the University of Cape Town, Cape Town, South Africa and Department of Rheumatology, Red Cross Children's Hospital, Cape Town, South Africa.; Institute for Experimental Endocrinology, Charite University Hospital, Berlin, Germany.; Department of Pediatrics, Institute of Medical Sciences, Medical College of Rzeszow University, University of Rzeszow, Rzeszow, Poland.; Department of Human Immunology, Institute of Medical Sciences, Medical College of Rzeszow University, University of Rzeszow, Rzeszow, Poland.; Department of Medicine, Mayo Clinic College of Medicine, Mayo Clinic, Rochester, MN, USA.

Publikováno v: Clinical orthopaedics and related research [Clin Orthop Relat Res] 2023 Dec 01; Vol. 481 (12), pp. 2447-2458. Date of Electronic Publication: 2023 May 08.

Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles.

Autor: Eekhoff EMW; Section Endocrinology, Department of Internal Medicine, Amsterdam University Medical Centers, Vrije Universiteit Amsterdam, Amsterdam Rare Bone Disease Center, Amsterdam Bone Center, Amsterdam Movement Sciences, Amsterdam, The Netherlands., de Ruiter RD; Section Endocrinology, Department of Internal Medicine, Amsterdam University Medical Centers, Vrije Universiteit Amsterdam, Amsterdam Rare Bone Disease Center, Amsterdam Bone Center, Amsterdam Movement Sciences, Amsterdam, The Netherlands., Smilde BJ; Section Endocrinology, Department of Internal Medicine, Amsterdam University Medical Centers, Vrije Universiteit Amsterdam, Amsterdam Rare Bone Disease Center, Amsterdam Bone Center, Amsterdam Movement Sciences, Amsterdam, The Netherlands., Schoenmaker T; Department of Periodontology, Academic Centre for Dentistry Amsterdam (ACTA), University of Amsterdam, Vrije Universiteit, Amsterdam, The Netherlands., de Vries TJ; Department of Periodontology, Academic Centre for Dentistry Amsterdam (ACTA), University of Amsterdam, Vrije Universiteit, Amsterdam, The Netherlands., Netelenbos C; Section Endocrinology, Department of Internal Medicine, Amsterdam University Medical Centers, Vrije Universiteit Amsterdam, Amsterdam Rare Bone Disease Center, Amsterdam Bone Center, Amsterdam Movement Sciences, Amsterdam, The Netherlands., Hsiao EC; Division of Endocrinology and Metabolism, Department of Medicine, Institute for Human Genetics, Program in Craniofacial Biology, the Institute for Regeneration Medicine, University of California, San Francisco, California, USA., Scott C; Division of Paediatric Rheumatology, Department of Paediatrics and Child Heath, Red Cross War Memorial Children's Hospital, University of Cape Town, Cape Town, South Africa., Haga N; Rehabilitation Services Bureau, National Rehabilitation Center for Persons with Disabilities, Tokorozawa, Saitama, Japan., Grunwald Z; Department of Anesthesiology, Sidney Kimmel Medical College at Thomas Jefferson University, Philadelphia, USA., De Cunto CL; Department of Pediatrics, Section Pediatric Rheumatology, Hospital Italiano Buenos Aires, Argentina., di Rocco M; Department of Pediatrics, Unit of Rare Diseases, IRCCS Giannina Gaslini Institute, Genoa, Italy., Delai PLR; Teaching and Research Institute of the Hospital Israelita Albert Einstein, Sao Paulo, Brazil., Diecidue RJ; Department of Oral and Maxillofacial Surgery, Sidney Kimmel Medical College, Philadelphia, USA., Madhuri V; Department of Paediatric Orthopaedics and Center for Stem Cell Research, Christian Medical College and Hospital, Vellore, India., Cho TJ; Department of Orthopaedic Surgery, Seoul National University College of Medicine, Seoul, Republic of Korea., Morhart R; Department of Pediatrics, Garmisch-Partenkichen Medical Center, Garmisch-Partenkirchen, Germany., Friedman CS; Schulich School of Medicine and Dentistry, Western University, Clinical Skills Building, London, Ontario, Canada., Zasloff M; Surgery and Pediatrics, MedStar Georgetown Transplant Institute, Washington, District of Columbia, USA., Pals G; Department of Human Genetics, Amsterdam University Medical Centers, Vrije Universiteit Amsterdam, Amsterdam Rare Bone Disease Center, Amsterdam Bone Center, Amsterdam Movement Sciences, Amsterdam, The Netherlands., Shim JH; Department of Medicine/Rheumatology, Horae Gene Therapy Center, Li Weibo Institute for Rare Diseases Research, UMass Chan Medical School, Worcester, Massachusetts, USA., Gao G; Department of Microbiology and Physiological Systems, Horae Gene Therapy Center, Viral Vector Core, Li Weibo Institute for Rare Diseases Research, UMass Chan Medical School, Worcester, Massachusetts, USA., Kaplan F; Department of Orthopaedic Surgery and Medicine, Center for Research in FOP and Related Disorders, The Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA., Pignolo RJ; Department of Medicine, Mayo Clinic, Rochester, Minnesota, USA., Micha D; Department of Human Genetics, Amsterdam University Medical Centers, Vrije Universiteit Amsterdam, Amsterdam Rare Bone Disease Center, Amsterdam Bone Center, Amsterdam Movement Sciences, Amsterdam, The Netherlands.

Publikováno v: Human gene therapy [Hum Gene Ther] 2022 Aug; Vol. 33 (15-16), pp. 782-788.

Spatial patterns of heterotopic ossification in fibrodysplasia ossificans progressiva correlate with anatomic temperature gradients.

Autor: Wang H; Department of Physiology and Biomedical Engineering, Mayo Clinic College of Medicine, Mayo Clinic, Rochester, MN 55905, USA; Department of Medicine, Mayo Clinic College of Medicine, Mayo Clinic, Rochester, MN, USA., De Cunto CL; Department of Pediatrics, Pediatric Rheumatology Section, Hospital Italiano de Buenos Aires, Gascón 450, 1181 Ciudad Autónoma de Buenos Aires, Argentina., Pignolo RJ; Department of Physiology and Biomedical Engineering, Mayo Clinic College of Medicine, Mayo Clinic, Rochester, MN 55905, USA; Department of Medicine, Mayo Clinic College of Medicine, Mayo Clinic, Rochester, MN, USA. Electronic address: Pignolo.Robert@mayo.edu., Kaplan FS; Department of Orthopaedic Surgery, The Perelman School of Medicine of The University of Pennsylvania, Philadelphia, PA 19104, USA; Department of Medicine, The Perelman School of Medicine of The University of Pennsylvania, Philadelphia, PA 19104, USA; Department of the Center for Research in FOP & Related Disorders, The Perelman School of Medicine of The University of Pennsylvania, Philadelphia, PA 19104, USA. Electronic address: Frederick.Kaplan@pennmedicine.upenn.edu.

Publikováno v: Bone [Bone] 2021 Aug; Vol. 149, pp. 115978. Date of Electronic Publication: 2021 Apr 27.

[Approach to the hidden curriculum through narrative medicine in the training of medical students].

Autor: Cacchiarelli San Román N; Servicio de Clínica Pediátrica del Hospital Italiano de Buenos Aires.; Instituto Universitario del Hospital Italiano de Buenos Aires. nicolas.cacchiarelli@hiba.org.ar.; Universidad de Buenos Aires., Eymann A; Servicio de Clínica Pediátrica del Hospital Italiano de Buenos Aires.; Instituto Universitario del Hospital Italiano de Buenos Aires.; Universidad de Buenos Aires., Roni C; Instituto Universitario del Hospital Italiano de Buenos Aires., De Cunto CL; Servicio de Clínica Pediátrica del Hospital Italiano de Buenos Aires.; Instituto Universitario del Hospital Italiano de Buenos Aires.

Publikováno v: Archivos argentinos de pediatria [Arch Argent Pediatr] 2020 Aug; Vol. 118 (4), pp. 290-294.