Phase 3 Trial of Oral Infigratinib in Children with Achondroplasia.

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Title: Phase 3 Trial of Oral Infigratinib in Children with Achondroplasia.
Authors: Savarirayan R; Murdoch Children's Research Institute, Melbourne, VIC, Australia., Hoover-Fong J; Johns Hopkins University, Baltimore., Irving M; Guy's and St. Thomas' NHS Foundation Trust, London., Arundel P; Sheffield Children's NHS Foundation Trust, Sheffield, United Kingdom., de Bergua JM; Arthroscopic Surgery Unit, Medical Innovation Knowledge Sharing Hospital, Vitoria-Gasteiz, Spain., Campeau PM; Centre Hospitalier Universitaire Sainte-Justine, Montreal., Candler T; University Hospitals Bristol and Weston NHS Foundation Trust, Bristol, United Kingdom., Cocanougher BT; Division of Human Genetics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati., Cormier-Daire V; Hôpital Necker-Enfants Malades, Université Paris Cité, INSERM Unité Mixte de Recherche 1163, Institut Imagine, Paris., Edouard T; Endocrine, Bone Diseases, and Genetics Unit, Reference Center for Rare Diseases of Calcium and Phosphate Metabolism, Pediatric Research Unit Centre d'Investigation Clinique 1436, Children's Hospital, Toulouse University Hospital, Toulouse, France., Fredwall SO; Oslo University Hospital, Oslo.; Norwegian Center for Rare Diseases, Unit Sunnaas, Nesodden, Norway., Harmatz P; UCSF Benioff Children's Hospital Oakland, Oakland, CA., Hoernschemeyer D; University of Missouri, Columbia., Irgens HU; Haukeland University Hospital, Bergen, Norway., Jamuar S; KK Women's and Children's Hospital, Singapore., Kannu P; University of Alberta-Stollery Children's Hospital, Edmonton, Canada., Legare JM; University of Wisconsin School of Medicine and Public Health, Madison., Leiva-Gea A; Hospital Universitario Virgen de la Victoria, Instituto de Investigación Biomédica de Málaga, Plataforma Bionand, Hospital Quironsalud Málaga, and Department of Biomedicine, Faculty of Biomedical Sciences and Sports, Universidad Europea de Andalucía, Malaga, Spain., McDevitt H; NHS Greater Glasgow and Clyde, Glasgow, United Kingdom., Onesimo R; Università Cattolica del Sacro Cuore-Fondazione Policlinico A. Gemelli IRCCS, Rome., Phillips J 3rd; Vanderbilt Health, Nashville., Del Pino M; Hospital de Pediatría Garrahan, Buenos Aires., Robinson ME; Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa., Rossi M; Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon, France., Skae M; Manchester University NHS Foundation Trust and University of Manchester, Manchester, United Kingdom., Ward LM; Children's Hospital at London Health Sciences Centre, London, ON, Canada., White KK; Children's Hospital of Colorado, Aurora., Schmidt J; BridgeBio Pharma, San Francisco., Lystig T; BridgeBio Pharma, San Francisco., Salvatici A; BridgeBio Pharma, San Francisco., Bai Y; BridgeBio Pharma, San Francisco., Butler PW; BridgeBio Pharma, San Francisco., van Veenhuyzen D; BridgeBio Pharma, San Francisco., Rogoff D; BridgeBio Pharma, San Francisco.
Source: The New England journal of medicine [N Engl J Med] 2026 Jun 28. Date of Electronic Publication: 2026 Jun 28.
Publication Type: Journal Article
Journal Info: Publisher: Massachusetts Medical Society Country of Publication: United States NLM ID: 0255562 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1533-4406 (Electronic) Linking ISSN: 00284793 NLM ISO Abbreviation: N Engl J Med Subsets: MEDLINE
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  Data: <searchLink fieldCode="AU" term="%22Savarirayan+R%22">Savarirayan R</searchLink>; Murdoch Children's Research Institute, Melbourne, VIC, Australia.<br /><searchLink fieldCode="AU" term="%22Hoover-Fong+J%22">Hoover-Fong J</searchLink>; Johns Hopkins University, Baltimore.<br /><searchLink fieldCode="AU" term="%22Irving+M%22">Irving M</searchLink>; Guy's and St. Thomas' NHS Foundation Trust, London.<br /><searchLink fieldCode="AU" term="%22Arundel+P%22">Arundel P</searchLink>; Sheffield Children's NHS Foundation Trust, Sheffield, United Kingdom.<br /><searchLink fieldCode="AU" term="%22de+Bergua+JM%22">de Bergua JM</searchLink>; Arthroscopic Surgery Unit, Medical Innovation Knowledge Sharing Hospital, Vitoria-Gasteiz, Spain.<br /><searchLink fieldCode="AU" term="%22Campeau+PM%22">Campeau PM</searchLink>; Centre Hospitalier Universitaire Sainte-Justine, Montreal.<br /><searchLink fieldCode="AU" term="%22Candler+T%22">Candler T</searchLink>; University Hospitals Bristol and Weston NHS Foundation Trust, Bristol, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Cocanougher+BT%22">Cocanougher BT</searchLink>; Division of Human Genetics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati.<br /><searchLink fieldCode="AU" term="%22Cormier-Daire+V%22">Cormier-Daire V</searchLink>; Hôpital Necker-Enfants Malades, Université Paris Cité, INSERM Unité Mixte de Recherche 1163, Institut Imagine, Paris.<br /><searchLink fieldCode="AU" term="%22Edouard+T%22">Edouard T</searchLink>; Endocrine, Bone Diseases, and Genetics Unit, Reference Center for Rare Diseases of Calcium and Phosphate Metabolism, Pediatric Research Unit Centre d'Investigation Clinique 1436, Children's Hospital, Toulouse University Hospital, Toulouse, France.<br /><searchLink fieldCode="AU" term="%22Fredwall+SO%22">Fredwall SO</searchLink>; Oslo University Hospital, Oslo.; Norwegian Center for Rare Diseases, Unit Sunnaas, Nesodden, Norway.<br /><searchLink fieldCode="AU" term="%22Harmatz+P%22">Harmatz P</searchLink>; UCSF Benioff Children's Hospital Oakland, Oakland, CA.<br /><searchLink fieldCode="AU" term="%22Hoernschemeyer+D%22">Hoernschemeyer D</searchLink>; University of Missouri, Columbia.<br /><searchLink fieldCode="AU" term="%22Irgens+HU%22">Irgens HU</searchLink>; Haukeland University Hospital, Bergen, Norway.<br /><searchLink fieldCode="AU" term="%22Jamuar+S%22">Jamuar S</searchLink>; KK Women's and Children's Hospital, Singapore.<br /><searchLink fieldCode="AU" term="%22Kannu+P%22">Kannu P</searchLink>; University of Alberta-Stollery Children's Hospital, Edmonton, Canada.<br /><searchLink fieldCode="AU" term="%22Legare+JM%22">Legare JM</searchLink>; University of Wisconsin School of Medicine and Public Health, Madison.<br /><searchLink fieldCode="AU" term="%22Leiva-Gea+A%22">Leiva-Gea A</searchLink>; Hospital Universitario Virgen de la Victoria, Instituto de Investigación Biomédica de Málaga, Plataforma Bionand, Hospital Quironsalud Málaga, and Department of Biomedicine, Faculty of Biomedical Sciences and Sports, Universidad Europea de Andalucía, Malaga, Spain.<br /><searchLink fieldCode="AU" term="%22McDevitt+H%22">McDevitt H</searchLink>; NHS Greater Glasgow and Clyde, Glasgow, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Onesimo+R%22">Onesimo R</searchLink>; Università Cattolica del Sacro Cuore-Fondazione Policlinico A. Gemelli IRCCS, Rome.<br /><searchLink fieldCode="AU" term="%22Phillips+J+3rd%22">Phillips J 3rd</searchLink>; Vanderbilt Health, Nashville.<br /><searchLink fieldCode="AU" term="%22Del+Pino+M%22">Del Pino M</searchLink>; Hospital de Pediatría Garrahan, Buenos Aires.<br /><searchLink fieldCode="AU" term="%22Robinson+ME%22">Robinson ME</searchLink>; Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa.<br /><searchLink fieldCode="AU" term="%22Rossi+M%22">Rossi M</searchLink>; Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon, France.<br /><searchLink fieldCode="AU" term="%22Skae+M%22">Skae M</searchLink>; Manchester University NHS Foundation Trust and University of Manchester, Manchester, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Ward+LM%22">Ward LM</searchLink>; Children's Hospital at London Health Sciences Centre, London, ON, Canada.<br /><searchLink fieldCode="AU" term="%22White+KK%22">White KK</searchLink>; Children's Hospital of Colorado, Aurora.<br /><searchLink fieldCode="AU" term="%22Schmidt+J%22">Schmidt J</searchLink>; BridgeBio Pharma, San Francisco.<br /><searchLink fieldCode="AU" term="%22Lystig+T%22">Lystig T</searchLink>; BridgeBio Pharma, San Francisco.<br /><searchLink fieldCode="AU" term="%22Salvatici+A%22">Salvatici A</searchLink>; BridgeBio Pharma, San Francisco.<br /><searchLink fieldCode="AU" term="%22Bai+Y%22">Bai Y</searchLink>; BridgeBio Pharma, San Francisco.<br /><searchLink fieldCode="AU" term="%22Butler+PW%22">Butler PW</searchLink>; BridgeBio Pharma, San Francisco.<br /><searchLink fieldCode="AU" term="%22van+Veenhuyzen+D%22">van Veenhuyzen D</searchLink>; BridgeBio Pharma, San Francisco.<br /><searchLink fieldCode="AU" term="%22Rogoff+D%22">Rogoff D</searchLink>; BridgeBio Pharma, San Francisco.
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  Data: <searchLink fieldCode="JN" term="%220255562%22">The New England journal of medicine</searchLink> [N Engl J Med] 2026 Jun 28. <i>Date of Electronic Publication: </i>2026 Jun 28.
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