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Life-Saving, Dose-Adjusted, Targeted Therapy in a Patient with a STAT3 Gain-of-Function Mutation.
Sarfati, Eytan; Hadjadj, Jérome; Fusaro, Mathieu; Klifa, Roman; Grimaud, Marion; Berteloot, Laureline; Hadchouel, Alice; Godot, Cécile; Stolzenberg, Marie-Claude; Frémond, Marie-Louise; Pressiat, Claire; Molina, Thierry; Fischer, Alain; Picard, Capucine; Renolleau, Sylvain; Rieux-Laucat, Frederic; Blanche, Stephane; Neven, Benedicte.
Afiliação
  • Sarfati E; Pediatric Immunohematology and Rheumatology Department, Hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (AP-HP), 149 rue de Sèvres, F-75015, Paris, France.
  • Hadjadj J; University of Paris, Paris, France.
  • Fusaro M; Laboratory of immunogenetics of pediatric autoimmune disease, Institut Imagine, INSERM UMR1163, Paris, France.
  • Klifa R; University of Paris, Paris, France.
  • Grimaud M; Study Center for Primary Immunodeficiencies, Necker-Enfants Malades, AP-HP centre, Paris, France.
  • Berteloot L; Pediatric Immunohematology and Rheumatology Department, Hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (AP-HP), 149 rue de Sèvres, F-75015, Paris, France.
  • Hadchouel A; University of Paris, Paris, France.
  • Godot C; Pediatric Intensive Care Unit, Necker-Enfants-Malades University Hospital, AP-HP centre, Paris, France.
  • Stolzenberg MC; Pediatric Radiology Unit, Hôpital Necker-Enfants Malades, AP-HP Centre, Paris, France.
  • Frémond ML; University of Paris, Paris, France.
  • Pressiat C; Pediatric Pneumology Unit, Hôpital Necker-Enfants Malades, AP-HP Centre, Paris, France.
  • Molina T; Pediatric Endocrinology Unit, Hôpital Necker-Enfants Malades, AP-HP Centre, Paris, France.
  • Fischer A; Laboratory of immunogenetics of pediatric autoimmune disease, Institut Imagine, INSERM UMR1163, Paris, France.
  • Picard C; University of Paris, Paris, France.
  • Renolleau S; Laboratory of Neurogenetics and Neuroinflammation, Institut Imagine, INSERM UMR1163, Paris, France.
  • Rieux-Laucat F; Department of Pharmacology, Hôpital Henri Mondor, AP-HP, Paris, France.
  • Blanche S; University of Paris, Paris, France.
  • Neven B; Department of Pathology, Hôpital Necker-Enfants Malades, AP-HP Centre, Paris, France.
J Clin Immunol ; 41(4): 807-810, 2021 05.
Article em En | MEDLINE | ID: mdl-33428086

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Predisposição Genética para Doença / Fator de Transcrição STAT3 / Terapia de Alvo Molecular / Mutação com Ganho de Função / Doenças do Sistema Imunitário Idioma: En Ano de publicação: 2021 Tipo de documento: Article País de afiliação: França

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Predisposição Genética para Doença / Fator de Transcrição STAT3 / Terapia de Alvo Molecular / Mutação com Ganho de Função / Doenças do Sistema Imunitário Idioma: En Ano de publicação: 2021 Tipo de documento: Article País de afiliação: França