Oral Infigratinib Therapy in Children with Achondroplasia.

Autor: Savarirayan R; From Murdoch Children's Research Institute, Melbourne, VIC, Australia (R.S., S.R.); Hospital Vithas Vitoria, Vitoria-Gasteiz (J.M.D.B.), Hospital Universitario Virgen de la Victoria, Malaga (B.D., A.L.-G.), and Hospital Universitario La Paz, Madrid (M. Salcedo) - all in Spain; Sheffield Children's NHS Foundation Trust, Sheffield (P.A.), Birmingham Women's and Children's NHS Foundation Trust, Birmingham (V.S.), NHS Greater Glasgow and Clyde, Glasgow (H.M.), Manchester University NHS Foundation Trust, Manchester (M. Skae), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol (T.C.), and Guy's and St. Thomas' NHS Foundation Trust, London (M.I.) - all in the United Kingdom; Hôpital des Enfants-Toulouse, Toulouse (J.P.S.), Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon (M.N., M.R.), and Hôpital Necker-Enfants Malades, Paris (V.C.-D.) - all in France; the University of Alberta-Stollery Children's Hospital, Edmonton, Canada (P.K.); Vanderbilt University Medical Center, Nashville (J.P.); Cincinnati Children's Hospital Medical Center, Cincinnati (H.S.); Benioff Children's Hospital Oakland, Oakland (P.H.), and BridgeBio Pharma, San Francisco (D.H., E.M., R.W., Y.B., D.R.) - both in California; and Johns Hopkins University, Baltimore (J.H.-F.)., De Bergua JM; From Murdoch Children's Research Institute, Melbourne, VIC, Australia (R.S., S.R.); Hospital Vithas Vitoria, Vitoria-Gasteiz (J.M.D.B.), Hospital Universitario Virgen de la Victoria, Malaga (B.D., A.L.-G.), and Hospital Universitario La Paz, Madrid (M. Salcedo) - all in Spain; Sheffield Children's NHS Foundation Trust, Sheffield (P.A.), Birmingham Women's and Children's NHS Foundation Trust, Birmingham (V.S.), NHS Greater Glasgow and Clyde, Glasgow (H.M.), Manchester University NHS Foundation Trust, Manchester (M. Skae), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol (T.C.), and Guy's and St. Thomas' NHS Foundation Trust, London (M.I.) - all in the United Kingdom; Hôpital des Enfants-Toulouse, Toulouse (J.P.S.), Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon (M.N., M.R.), and Hôpital Necker-Enfants Malades, Paris (V.C.-D.) - all in France; the University of Alberta-Stollery Children's Hospital, Edmonton, Canada (P.K.); Vanderbilt University Medical Center, Nashville (J.P.); Cincinnati Children's Hospital Medical Center, Cincinnati (H.S.); Benioff Children's Hospital Oakland, Oakland (P.H.), and BridgeBio Pharma, San Francisco (D.H., E.M., R.W., Y.B., D.R.) - both in California; and Johns Hopkins University, Baltimore (J.H.-F.)., Arundel P; From Murdoch Children's Research Institute, Melbourne, VIC, Australia (R.S., S.R.); Hospital Vithas Vitoria, Vitoria-Gasteiz (J.M.D.B.), Hospital Universitario Virgen de la Victoria, Malaga (B.D., A.L.-G.), and Hospital Universitario La Paz, Madrid (M. Salcedo) - all in Spain; Sheffield Children's NHS Foundation Trust, Sheffield (P.A.), Birmingham Women's and Children's NHS Foundation Trust, Birmingham (V.S.), NHS Greater Glasgow and Clyde, Glasgow (H.M.), Manchester University NHS Foundation Trust, Manchester (M. Skae), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol (T.C.), and Guy's and St. Thomas' NHS Foundation Trust, London (M.I.) - all in the United Kingdom; Hôpital des Enfants-Toulouse, Toulouse (J.P.S.), Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon (M.N., M.R.), and Hôpital Necker-Enfants Malades, Paris (V.C.-D.) - all in France; the University of Alberta-Stollery Children's Hospital, Edmonton, Canada (P.K.); Vanderbilt University Medical Center, Nashville (J.P.); Cincinnati Children's Hospital Medical Center, Cincinnati (H.S.); Benioff Children's Hospital Oakland, Oakland (P.H.), and BridgeBio Pharma, San Francisco (D.H., E.M., R.W., Y.B., D.R.) - both in California; and Johns Hopkins University, Baltimore (J.H.-F.)., Salles JP; From Murdoch Children's Research Institute, Melbourne, VIC, Australia (R.S., S.R.); Hospital Vithas Vitoria, Vitoria-Gasteiz (J.M.D.B.), Hospital Universitario Virgen de la Victoria, Malaga (B.D., A.L.-G.), and Hospital Universitario La Paz, Madrid (M. Salcedo) - all in Spain; Sheffield Children's NHS Foundation Trust, Sheffield (P.A.), Birmingham Women's and Children's NHS Foundation Trust, Birmingham (V.S.), NHS Greater Glasgow and Clyde, Glasgow (H.M.), Manchester University NHS Foundation Trust, Manchester (M. Skae), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol (T.C.), and Guy's and St. Thomas' NHS Foundation Trust, London (M.I.) - all in the United Kingdom; Hôpital des Enfants-Toulouse, Toulouse (J.P.S.), Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon (M.N., M.R.), and Hôpital Necker-Enfants Malades, Paris (V.C.-D.) - all in France; the University of Alberta-Stollery Children's Hospital, Edmonton, Canada (P.K.); Vanderbilt University Medical Center, Nashville (J.P.); Cincinnati Children's Hospital Medical Center, Cincinnati (H.S.); Benioff Children's Hospital Oakland, Oakland (P.H.), and BridgeBio Pharma, San Francisco (D.H., E.M., R.W., Y.B., D.R.) - both in California; and Johns Hopkins University, Baltimore (J.H.-F.)., Saraff V; From Murdoch Children's Research Institute, Melbourne, VIC, Australia (R.S., S.R.); Hospital Vithas Vitoria, Vitoria-Gasteiz (J.M.D.B.), Hospital Universitario Virgen de la Victoria, Malaga (B.D., A.L.-G.), and Hospital Universitario La Paz, Madrid (M. Salcedo) - all in Spain; Sheffield Children's NHS Foundation Trust, Sheffield (P.A.), Birmingham Women's and Children's NHS Foundation Trust, Birmingham (V.S.), NHS Greater Glasgow and Clyde, Glasgow (H.M.), Manchester University NHS Foundation Trust, Manchester (M. Skae), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol (T.C.), and Guy's and St. Thomas' NHS Foundation Trust, London (M.I.) - all in the United Kingdom; Hôpital des Enfants-Toulouse, Toulouse (J.P.S.), Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon (M.N., M.R.), and Hôpital Necker-Enfants Malades, Paris (V.C.-D.) - all in France; the University of Alberta-Stollery Children's Hospital, Edmonton, Canada (P.K.); Vanderbilt University Medical Center, Nashville (J.P.); Cincinnati Children's Hospital Medical Center, Cincinnati (H.S.); Benioff Children's Hospital Oakland, Oakland (P.H.), and BridgeBio Pharma, San Francisco (D.H., E.M., R.W., Y.B., D.R.) - both in California; and Johns Hopkins University, Baltimore (J.H.-F.)., Delgado B; From Murdoch Children's Research Institute, Melbourne, VIC, Australia (R.S., S.R.); Hospital Vithas Vitoria, Vitoria-Gasteiz (J.M.D.B.), Hospital Universitario Virgen de la Victoria, Malaga (B.D., A.L.-G.), and Hospital Universitario La Paz, Madrid (M. Salcedo) - all in Spain; Sheffield Children's NHS Foundation Trust, Sheffield (P.A.), Birmingham Women's and Children's NHS Foundation Trust, Birmingham (V.S.), NHS Greater Glasgow and Clyde, Glasgow (H.M.), Manchester University NHS Foundation Trust, Manchester (M. Skae), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol (T.C.), and Guy's and St. Thomas' NHS Foundation Trust, London (M.I.) - all in the United Kingdom; Hôpital des Enfants-Toulouse, Toulouse (J.P.S.), Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon (M.N., M.R.), and Hôpital Necker-Enfants Malades, Paris (V.C.-D.) - all in France; the University of Alberta-Stollery Children's Hospital, Edmonton, Canada (P.K.); Vanderbilt University Medical Center, Nashville (J.P.); Cincinnati Children's Hospital Medical Center, Cincinnati (H.S.); Benioff Children's Hospital Oakland, Oakland (P.H.), and BridgeBio Pharma, San Francisco (D.H., E.M., R.W., Y.B., D.R.) - both in California; and Johns Hopkins University, Baltimore (J.H.-F.)., Leiva-Gea A; From Murdoch Children's Research Institute, Melbourne, VIC, Australia (R.S., S.R.); Hospital Vithas Vitoria, Vitoria-Gasteiz (J.M.D.B.), Hospital Universitario Virgen de la Victoria, Malaga (B.D., A.L.-G.), and Hospital Universitario La Paz, Madrid (M. Salcedo) - all in Spain; Sheffield Children's NHS Foundation Trust, Sheffield (P.A.), Birmingham Women's and Children's NHS Foundation Trust, Birmingham (V.S.), NHS Greater Glasgow and Clyde, Glasgow (H.M.), Manchester University NHS Foundation Trust, Manchester (M. Skae), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol (T.C.), and Guy's and St. Thomas' NHS Foundation Trust, London (M.I.) - all in the United Kingdom; Hôpital des Enfants-Toulouse, Toulouse (J.P.S.), Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon (M.N., M.R.), and Hôpital Necker-Enfants Malades, Paris (V.C.-D.) - all in France; the University of Alberta-Stollery Children's Hospital, Edmonton, Canada (P.K.); Vanderbilt University Medical Center, Nashville (J.P.); Cincinnati Children's Hospital Medical Center, Cincinnati (H.S.); Benioff Children's Hospital Oakland, Oakland (P.H.), and BridgeBio Pharma, San Francisco (D.H., E.M., R.W., Y.B., D.R.) - both in California; and Johns Hopkins University, Baltimore (J.H.-F.)., McDevitt H; From Murdoch Children's Research Institute, Melbourne, VIC, Australia (R.S., S.R.); Hospital Vithas Vitoria, Vitoria-Gasteiz (J.M.D.B.), Hospital Universitario Virgen de la Victoria, Malaga (B.D., A.L.-G.), and Hospital Universitario La Paz, Madrid (M. Salcedo) - all in Spain; Sheffield Children's NHS Foundation Trust, Sheffield (P.A.), Birmingham Women's and Children's NHS Foundation Trust, Birmingham (V.S.), NHS Greater Glasgow and Clyde, Glasgow (H.M.), Manchester University NHS Foundation Trust, Manchester (M. Skae), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol (T.C.), and Guy's and St. Thomas' NHS Foundation Trust, London (M.I.) - all in the United Kingdom; Hôpital des Enfants-Toulouse, Toulouse (J.P.S.), Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon (M.N., M.R.), and Hôpital Necker-Enfants Malades, Paris (V.C.-D.) - all in France; the University of Alberta-Stollery Children's Hospital, Edmonton, Canada (P.K.); Vanderbilt University Medical Center, Nashville (J.P.); Cincinnati Children's Hospital Medical Center, Cincinnati (H.S.); Benioff Children's Hospital Oakland, Oakland (P.H.), and BridgeBio Pharma, San Francisco (D.H., E.M., R.W., Y.B., D.R.) - both in California; and Johns Hopkins University, Baltimore (J.H.-F.)., Nicolino M; From Murdoch Children's Research Institute, Melbourne, VIC, Australia (R.S., S.R.); Hospital Vithas Vitoria, Vitoria-Gasteiz (J.M.D.B.), Hospital Universitario Virgen de la Victoria, Malaga (B.D., A.L.-G.), and Hospital Universitario La Paz, Madrid (M. Salcedo) - all in Spain; Sheffield Children's NHS Foundation Trust, Sheffield (P.A.), Birmingham Women's and Children's NHS Foundation Trust, Birmingham (V.S.), NHS Greater Glasgow and Clyde, Glasgow (H.M.), Manchester University NHS Foundation Trust, Manchester (M. Skae), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol (T.C.), and Guy's and St. Thomas' NHS Foundation Trust, London (M.I.) - all in the United Kingdom; Hôpital des Enfants-Toulouse, Toulouse (J.P.S.), Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon (M.N., M.R.), and Hôpital Necker-Enfants Malades, Paris (V.C.-D.) - all in France; the University of Alberta-Stollery Children's Hospital, Edmonton, Canada (P.K.); Vanderbilt University Medical Center, Nashville (J.P.); Cincinnati Children's Hospital Medical Center, Cincinnati (H.S.); Benioff Children's Hospital Oakland, Oakland (P.H.), and BridgeBio Pharma, San Francisco (D.H., E.M., R.W., Y.B., D.R.) - both in California; and Johns Hopkins University, Baltimore (J.H.-F.)., Rossi M; From Murdoch Children's Research Institute, Melbourne, VIC, Australia (R.S., S.R.); Hospital Vithas Vitoria, Vitoria-Gasteiz (J.M.D.B.), Hospital Universitario Virgen de la Victoria, Malaga (B.D., A.L.-G.), and Hospital Universitario La Paz, Madrid (M. Salcedo) - all in Spain; Sheffield Children's NHS Foundation Trust, Sheffield (P.A.), Birmingham Women's and Children's NHS Foundation Trust, Birmingham (V.S.), NHS Greater Glasgow and Clyde, Glasgow (H.M.), Manchester University NHS Foundation Trust, Manchester (M. Skae), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol (T.C.), and Guy's and St. Thomas' NHS Foundation Trust, London (M.I.) - all in the United Kingdom; Hôpital des Enfants-Toulouse, Toulouse (J.P.S.), Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon (M.N., M.R.), and Hôpital Necker-Enfants Malades, Paris (V.C.-D.) - all in France; the University of Alberta-Stollery Children's Hospital, Edmonton, Canada (P.K.); Vanderbilt University Medical Center, Nashville (J.P.); Cincinnati Children's Hospital Medical Center, Cincinnati (H.S.); Benioff Children's Hospital Oakland, Oakland (P.H.), and BridgeBio Pharma, San Francisco (D.H., E.M., R.W., Y.B., D.R.) - both in California; and Johns Hopkins University, Baltimore (J.H.-F.)., Salcedo M; From Murdoch Children's Research Institute, Melbourne, VIC, Australia (R.S., S.R.); Hospital Vithas Vitoria, Vitoria-Gasteiz (J.M.D.B.), Hospital Universitario Virgen de la Victoria, Malaga (B.D., A.L.-G.), and Hospital Universitario La Paz, Madrid (M. Salcedo) - all in Spain; Sheffield Children's NHS Foundation Trust, Sheffield (P.A.), Birmingham Women's and Children's NHS Foundation Trust, Birmingham (V.S.), NHS Greater Glasgow and Clyde, Glasgow (H.M.), Manchester University NHS Foundation Trust, Manchester (M. Skae), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol (T.C.), and Guy's and St. Thomas' NHS Foundation Trust, London (M.I.) - all in the United Kingdom; Hôpital des Enfants-Toulouse, Toulouse (J.P.S.), Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon (M.N., M.R.), and Hôpital Necker-Enfants Malades, Paris (V.C.-D.) - all in France; the University of Alberta-Stollery Children's Hospital, Edmonton, Canada (P.K.); Vanderbilt University Medical Center, Nashville (J.P.); Cincinnati Children's Hospital Medical Center, Cincinnati (H.S.); Benioff Children's Hospital Oakland, Oakland (P.H.), and BridgeBio Pharma, San Francisco (D.H., E.M., R.W., Y.B., D.R.) - both in California; and Johns Hopkins University, Baltimore (J.H.-F.)., Cormier-Daire V; From Murdoch Children's Research Institute, Melbourne, VIC, Australia (R.S., S.R.); Hospital Vithas Vitoria, Vitoria-Gasteiz (J.M.D.B.), Hospital Universitario Virgen de la Victoria, Malaga (B.D., A.L.-G.), and Hospital Universitario La Paz, Madrid (M. Salcedo) - all in Spain; Sheffield Children's NHS Foundation Trust, Sheffield (P.A.), Birmingham Women's and Children's NHS Foundation Trust, Birmingham (V.S.), NHS Greater Glasgow and Clyde, Glasgow (H.M.), Manchester University NHS Foundation Trust, Manchester (M. Skae), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol (T.C.), and Guy's and St. Thomas' NHS Foundation Trust, London (M.I.) - all in the United Kingdom; Hôpital des Enfants-Toulouse, Toulouse (J.P.S.), Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon (M.N., M.R.), and Hôpital Necker-Enfants Malades, Paris (V.C.-D.) - all in France; the University of Alberta-Stollery Children's Hospital, Edmonton, Canada (P.K.); Vanderbilt University Medical Center, Nashville (J.P.); Cincinnati Children's Hospital Medical Center, Cincinnati (H.S.); Benioff Children's Hospital Oakland, Oakland (P.H.), and BridgeBio Pharma, San Francisco (D.H., E.M., R.W., Y.B., D.R.) - both in California; and Johns Hopkins University, Baltimore (J.H.-F.)., Skae M; From Murdoch Children's Research Institute, Melbourne, VIC, Australia (R.S., S.R.); Hospital Vithas Vitoria, Vitoria-Gasteiz (J.M.D.B.), Hospital Universitario Virgen de la Victoria, Malaga (B.D., A.L.-G.), and Hospital Universitario La Paz, Madrid (M. Salcedo) - all in Spain; Sheffield Children's NHS Foundation Trust, Sheffield (P.A.), Birmingham Women's and Children's NHS Foundation Trust, Birmingham (V.S.), NHS Greater Glasgow and Clyde, Glasgow (H.M.), Manchester University NHS Foundation Trust, Manchester (M. Skae), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol (T.C.), and Guy's and St. Thomas' NHS Foundation Trust, London (M.I.) - all in the United Kingdom; Hôpital des Enfants-Toulouse, Toulouse (J.P.S.), Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon (M.N., M.R.), and Hôpital Necker-Enfants Malades, Paris (V.C.-D.) - all in France; the University of Alberta-Stollery Children's Hospital, Edmonton, Canada (P.K.); Vanderbilt University Medical Center, Nashville (J.P.); Cincinnati Children's Hospital Medical Center, Cincinnati (H.S.); Benioff Children's Hospital Oakland, Oakland (P.H.), and BridgeBio Pharma, San Francisco (D.H., E.M., R.W., Y.B., D.R.) - both in California; and Johns Hopkins University, Baltimore (J.H.-F.)., Kannu P; From Murdoch Children's Research Institute, Melbourne, VIC, Australia (R.S., S.R.); Hospital Vithas Vitoria, Vitoria-Gasteiz (J.M.D.B.), Hospital Universitario Virgen de la Victoria, Malaga (B.D., A.L.-G.), and Hospital Universitario La Paz, Madrid (M. Salcedo) - all in Spain; Sheffield Children's NHS Foundation Trust, Sheffield (P.A.), Birmingham Women's and Children's NHS Foundation Trust, Birmingham (V.S.), NHS Greater Glasgow and Clyde, Glasgow (H.M.), Manchester University NHS Foundation Trust, Manchester (M. Skae), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol (T.C.), and Guy's and St. Thomas' NHS Foundation Trust, London (M.I.) - all in the United Kingdom; Hôpital des Enfants-Toulouse, Toulouse (J.P.S.), Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon (M.N., M.R.), and Hôpital Necker-Enfants Malades, Paris (V.C.-D.) - all in France; the University of Alberta-Stollery Children's Hospital, Edmonton, Canada (P.K.); Vanderbilt University Medical Center, Nashville (J.P.); Cincinnati Children's Hospital Medical Center, Cincinnati (H.S.); Benioff Children's Hospital Oakland, Oakland (P.H.), and BridgeBio Pharma, San Francisco (D.H., E.M., R.W., Y.B., D.R.) - both in California; and Johns Hopkins University, Baltimore (J.H.-F.)., Phillips J 3rd; From Murdoch Children's Research Institute, Melbourne, VIC, Australia (R.S., S.R.); Hospital Vithas Vitoria, Vitoria-Gasteiz (J.M.D.B.), Hospital Universitario Virgen de la Victoria, Malaga (B.D., A.L.-G.), and Hospital Universitario La Paz, Madrid (M. Salcedo) - all in Spain; Sheffield Children's NHS Foundation Trust, Sheffield (P.A.), Birmingham Women's and Children's NHS Foundation Trust, Birmingham (V.S.), NHS Greater Glasgow and Clyde, Glasgow (H.M.), Manchester University NHS Foundation Trust, Manchester (M. Skae), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol (T.C.), and Guy's and St. Thomas' NHS Foundation Trust, London (M.I.) - all in the United Kingdom; Hôpital des Enfants-Toulouse, Toulouse (J.P.S.), Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon (M.N., M.R.), and Hôpital Necker-Enfants Malades, Paris (V.C.-D.) - all in France; the University of Alberta-Stollery Children's Hospital, Edmonton, Canada (P.K.); Vanderbilt University Medical Center, Nashville (J.P.); Cincinnati Children's Hospital Medical Center, Cincinnati (H.S.); Benioff Children's Hospital Oakland, Oakland (P.H.), and BridgeBio Pharma, San Francisco (D.H., E.M., R.W., Y.B., D.R.) - both in California; and Johns Hopkins University, Baltimore (J.H.-F.)., Saal H; From Murdoch Children's Research Institute, Melbourne, VIC, Australia (R.S., S.R.); Hospital Vithas Vitoria, Vitoria-Gasteiz (J.M.D.B.), Hospital Universitario Virgen de la Victoria, Malaga (B.D., A.L.-G.), and Hospital Universitario La Paz, Madrid (M. Salcedo) - all in Spain; Sheffield Children's NHS Foundation Trust, Sheffield (P.A.), Birmingham Women's and Children's NHS Foundation Trust, Birmingham (V.S.), NHS Greater Glasgow and Clyde, Glasgow (H.M.), Manchester University NHS Foundation Trust, Manchester (M. Skae), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol (T.C.), and Guy's and St. Thomas' NHS Foundation Trust, London (M.I.) - all in the United Kingdom; Hôpital des Enfants-Toulouse, Toulouse (J.P.S.), Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon (M.N., M.R.), and Hôpital Necker-Enfants Malades, Paris (V.C.-D.) - all in France; the University of Alberta-Stollery Children's Hospital, Edmonton, Canada (P.K.); Vanderbilt University Medical Center, Nashville (J.P.); Cincinnati Children's Hospital Medical Center, Cincinnati (H.S.); Benioff Children's Hospital Oakland, Oakland (P.H.), and BridgeBio Pharma, San Francisco (D.H., E.M., R.W., Y.B., D.R.) - both in California; and Johns Hopkins University, Baltimore (J.H.-F.)., Harmatz P; From Murdoch Children's Research Institute, Melbourne, VIC, Australia (R.S., S.R.); Hospital Vithas Vitoria, Vitoria-Gasteiz (J.M.D.B.), Hospital Universitario Virgen de la Victoria, Malaga (B.D., A.L.-G.), and Hospital Universitario La Paz, Madrid (M. Salcedo) - all in Spain; Sheffield Children's NHS Foundation Trust, Sheffield (P.A.), Birmingham Women's and Children's NHS Foundation Trust, Birmingham (V.S.), NHS Greater Glasgow and Clyde, Glasgow (H.M.), Manchester University NHS Foundation Trust, Manchester (M. Skae), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol (T.C.), and Guy's and St. Thomas' NHS Foundation Trust, London (M.I.) - all in the United Kingdom; Hôpital des Enfants-Toulouse, Toulouse (J.P.S.), Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon (M.N., M.R.), and Hôpital Necker-Enfants Malades, Paris (V.C.-D.) - all in France; the University of Alberta-Stollery Children's Hospital, Edmonton, Canada (P.K.); Vanderbilt University Medical Center, Nashville (J.P.); Cincinnati Children's Hospital Medical Center, Cincinnati (H.S.); Benioff Children's Hospital Oakland, Oakland (P.H.), and BridgeBio Pharma, San Francisco (D.H., E.M., R.W., Y.B., D.R.) - both in California; and Johns Hopkins University, Baltimore (J.H.-F.)., Candler T; From Murdoch Children's Research Institute, Melbourne, VIC, Australia (R.S., S.R.); Hospital Vithas Vitoria, Vitoria-Gasteiz (J.M.D.B.), Hospital Universitario Virgen de la Victoria, Malaga (B.D., A.L.-G.), and Hospital Universitario La Paz, Madrid (M. Salcedo) - all in Spain; Sheffield Children's NHS Foundation Trust, Sheffield (P.A.), Birmingham Women's and Children's NHS Foundation Trust, Birmingham (V.S.), NHS Greater Glasgow and Clyde, Glasgow (H.M.), Manchester University NHS Foundation Trust, Manchester (M. Skae), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol (T.C.), and Guy's and St. Thomas' NHS Foundation Trust, London (M.I.) - all in the United Kingdom; Hôpital des Enfants-Toulouse, Toulouse (J.P.S.), Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon (M.N., M.R.), and Hôpital Necker-Enfants Malades, Paris (V.C.-D.) - all in France; the University of Alberta-Stollery Children's Hospital, Edmonton, Canada (P.K.); Vanderbilt University Medical Center, Nashville (J.P.); Cincinnati Children's Hospital Medical Center, Cincinnati (H.S.); Benioff Children's Hospital Oakland, Oakland (P.H.), and BridgeBio Pharma, San Francisco (D.H., E.M., R.W., Y.B., D.R.) - both in California; and Johns Hopkins University, Baltimore (J.H.-F.)., Hill D; From Murdoch Children's Research Institute, Melbourne, VIC, Australia (R.S., S.R.); Hospital Vithas Vitoria, Vitoria-Gasteiz (J.M.D.B.), Hospital Universitario Virgen de la Victoria, Malaga (B.D., A.L.-G.), and Hospital Universitario La Paz, Madrid (M. Salcedo) - all in Spain; Sheffield Children's NHS Foundation Trust, Sheffield (P.A.), Birmingham Women's and Children's NHS Foundation Trust, Birmingham (V.S.), NHS Greater Glasgow and Clyde, Glasgow (H.M.), Manchester University NHS Foundation Trust, Manchester (M. Skae), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol (T.C.), and Guy's and St. Thomas' NHS Foundation Trust, London (M.I.) - all in the United Kingdom; Hôpital des Enfants-Toulouse, Toulouse (J.P.S.), Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon (M.N., M.R.), and Hôpital Necker-Enfants Malades, Paris (V.C.-D.) - all in France; the University of Alberta-Stollery Children's Hospital, Edmonton, Canada (P.K.); Vanderbilt University Medical Center, Nashville (J.P.); Cincinnati Children's Hospital Medical Center, Cincinnati (H.S.); Benioff Children's Hospital Oakland, Oakland (P.H.), and BridgeBio Pharma, San Francisco (D.H., E.M., R.W., Y.B., D.R.) - both in California; and Johns Hopkins University, Baltimore (J.H.-F.)., Muslimova E; From Murdoch Children's Research Institute, Melbourne, VIC, Australia (R.S., S.R.); Hospital Vithas Vitoria, Vitoria-Gasteiz (J.M.D.B.), Hospital Universitario Virgen de la Victoria, Malaga (B.D., A.L.-G.), and Hospital Universitario La Paz, Madrid (M. Salcedo) - all in Spain; Sheffield Children's NHS Foundation Trust, Sheffield (P.A.), Birmingham Women's and Children's NHS Foundation Trust, Birmingham (V.S.), NHS Greater Glasgow and Clyde, Glasgow (H.M.), Manchester University NHS Foundation Trust, Manchester (M. Skae), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol (T.C.), and Guy's and St. Thomas' NHS Foundation Trust, London (M.I.) - all in the United Kingdom; Hôpital des Enfants-Toulouse, Toulouse (J.P.S.), Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon (M.N., M.R.), and Hôpital Necker-Enfants Malades, Paris (V.C.-D.) - all in France; the University of Alberta-Stollery Children's Hospital, Edmonton, Canada (P.K.); Vanderbilt University Medical Center, Nashville (J.P.); Cincinnati Children's Hospital Medical Center, Cincinnati (H.S.); Benioff Children's Hospital Oakland, Oakland (P.H.), and BridgeBio Pharma, San Francisco (D.H., E.M., R.W., Y.B., D.R.) - both in California; and Johns Hopkins University, Baltimore (J.H.-F.)., Weng R; From Murdoch Children's Research Institute, Melbourne, VIC, Australia (R.S., S.R.); Hospital Vithas Vitoria, Vitoria-Gasteiz (J.M.D.B.), Hospital Universitario Virgen de la Victoria, Malaga (B.D., A.L.-G.), and Hospital Universitario La Paz, Madrid (M. Salcedo) - all in Spain; Sheffield Children's NHS Foundation Trust, Sheffield (P.A.), Birmingham Women's and Children's NHS Foundation Trust, Birmingham (V.S.), NHS Greater Glasgow and Clyde, Glasgow (H.M.), Manchester University NHS Foundation Trust, Manchester (M. Skae), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol (T.C.), and Guy's and St. Thomas' NHS Foundation Trust, London (M.I.) - all in the United Kingdom; Hôpital des Enfants-Toulouse, Toulouse (J.P.S.), Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon (M.N., M.R.), and Hôpital Necker-Enfants Malades, Paris (V.C.-D.) - all in France; the University of Alberta-Stollery Children's Hospital, Edmonton, Canada (P.K.); Vanderbilt University Medical Center, Nashville (J.P.); Cincinnati Children's Hospital Medical Center, Cincinnati (H.S.); Benioff Children's Hospital Oakland, Oakland (P.H.), and BridgeBio Pharma, San Francisco (D.H., E.M., R.W., Y.B., D.R.) - both in California; and Johns Hopkins University, Baltimore (J.H.-F.)., Bai Y; From Murdoch Children's Research Institute, Melbourne, VIC, Australia (R.S., S.R.); Hospital Vithas Vitoria, Vitoria-Gasteiz (J.M.D.B.), Hospital Universitario Virgen de la Victoria, Malaga (B.D., A.L.-G.), and Hospital Universitario La Paz, Madrid (M. Salcedo) - all in Spain; Sheffield Children's NHS Foundation Trust, Sheffield (P.A.), Birmingham Women's and Children's NHS Foundation Trust, Birmingham (V.S.), NHS Greater Glasgow and Clyde, Glasgow (H.M.), Manchester University NHS Foundation Trust, Manchester (M. Skae), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol (T.C.), and Guy's and St. Thomas' NHS Foundation Trust, London (M.I.) - all in the United Kingdom; Hôpital des Enfants-Toulouse, Toulouse (J.P.S.), Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon (M.N., M.R.), and Hôpital Necker-Enfants Malades, Paris (V.C.-D.) - all in France; the University of Alberta-Stollery Children's Hospital, Edmonton, Canada (P.K.); Vanderbilt University Medical Center, Nashville (J.P.); Cincinnati Children's Hospital Medical Center, Cincinnati (H.S.); Benioff Children's Hospital Oakland, Oakland (P.H.), and BridgeBio Pharma, San Francisco (D.H., E.M., R.W., Y.B., D.R.) - both in California; and Johns Hopkins University, Baltimore (J.H.-F.)., Raj S; From Murdoch Children's Research Institute, Melbourne, VIC, Australia (R.S., S.R.); Hospital Vithas Vitoria, Vitoria-Gasteiz (J.M.D.B.), Hospital Universitario Virgen de la Victoria, Malaga (B.D., A.L.-G.), and Hospital Universitario La Paz, Madrid (M. Salcedo) - all in Spain; Sheffield Children's NHS Foundation Trust, Sheffield (P.A.), Birmingham Women's and Children's NHS Foundation Trust, Birmingham (V.S.), NHS Greater Glasgow and Clyde, Glasgow (H.M.), Manchester University NHS Foundation Trust, Manchester (M. Skae), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol (T.C.), and Guy's and St. Thomas' NHS Foundation Trust, London (M.I.) - all in the United Kingdom; Hôpital des Enfants-Toulouse, Toulouse (J.P.S.), Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon (M.N., M.R.), and Hôpital Necker-Enfants Malades, Paris (V.C.-D.) - all in France; the University of Alberta-Stollery Children's Hospital, Edmonton, Canada (P.K.); Vanderbilt University Medical Center, Nashville (J.P.); Cincinnati Children's Hospital Medical Center, Cincinnati (H.S.); Benioff Children's Hospital Oakland, Oakland (P.H.), and BridgeBio Pharma, San Francisco (D.H., E.M., R.W., Y.B., D.R.) - both in California; and Johns Hopkins University, Baltimore (J.H.-F.)., Hoover-Fong J; From Murdoch Children's Research Institute, Melbourne, VIC, Australia (R.S., S.R.); Hospital Vithas Vitoria, Vitoria-Gasteiz (J.M.D.B.), Hospital Universitario Virgen de la Victoria, Malaga (B.D., A.L.-G.), and Hospital Universitario La Paz, Madrid (M. Salcedo) - all in Spain; Sheffield Children's NHS Foundation Trust, Sheffield (P.A.), Birmingham Women's and Children's NHS Foundation Trust, Birmingham (V.S.), NHS Greater Glasgow and Clyde, Glasgow (H.M.), Manchester University NHS Foundation Trust, Manchester (M. Skae), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol (T.C.), and Guy's and St. Thomas' NHS Foundation Trust, London (M.I.) - all in the United Kingdom; Hôpital des Enfants-Toulouse, Toulouse (J.P.S.), Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon (M.N., M.R.), and Hôpital Necker-Enfants Malades, Paris (V.C.-D.) - all in France; the University of Alberta-Stollery Children's Hospital, Edmonton, Canada (P.K.); Vanderbilt University Medical Center, Nashville (J.P.); Cincinnati Children's Hospital Medical Center, Cincinnati (H.S.); Benioff Children's Hospital Oakland, Oakland (P.H.), and BridgeBio Pharma, San Francisco (D.H., E.M., R.W., Y.B., D.R.) - both in California; and Johns Hopkins University, Baltimore (J.H.-F.)., Irving M; From Murdoch Children's Research Institute, Melbourne, VIC, Australia (R.S., S.R.); Hospital Vithas Vitoria, Vitoria-Gasteiz (J.M.D.B.), Hospital Universitario Virgen de la Victoria, Malaga (B.D., A.L.-G.), and Hospital Universitario La Paz, Madrid (M. Salcedo) - all in Spain; Sheffield Children's NHS Foundation Trust, Sheffield (P.A.), Birmingham Women's and Children's NHS Foundation Trust, Birmingham (V.S.), NHS Greater Glasgow and Clyde, Glasgow (H.M.), Manchester University NHS Foundation Trust, Manchester (M. Skae), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol (T.C.), and Guy's and St. Thomas' NHS Foundation Trust, London (M.I.) - all in the United Kingdom; Hôpital des Enfants-Toulouse, Toulouse (J.P.S.), Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon (M.N., M.R.), and Hôpital Necker-Enfants Malades, Paris (V.C.-D.) - all in France; the University of Alberta-Stollery Children's Hospital, Edmonton, Canada (P.K.); Vanderbilt University Medical Center, Nashville (J.P.); Cincinnati Children's Hospital Medical Center, Cincinnati (H.S.); Benioff Children's Hospital Oakland, Oakland (P.H.), and BridgeBio Pharma, San Francisco (D.H., E.M., R.W., Y.B., D.R.) - both in California; and Johns Hopkins University, Baltimore (J.H.-F.)., Rogoff D; From Murdoch Children's Research Institute, Melbourne, VIC, Australia (R.S., S.R.); Hospital Vithas Vitoria, Vitoria-Gasteiz (J.M.D.B.), Hospital Universitario Virgen de la Victoria, Malaga (B.D., A.L.-G.), and Hospital Universitario La Paz, Madrid (M. Salcedo) - all in Spain; Sheffield Children's NHS Foundation Trust, Sheffield (P.A.), Birmingham Women's and Children's NHS Foundation Trust, Birmingham (V.S.), NHS Greater Glasgow and Clyde, Glasgow (H.M.), Manchester University NHS Foundation Trust, Manchester (M. Skae), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol (T.C.), and Guy's and St. Thomas' NHS Foundation Trust, London (M.I.) - all in the United Kingdom; Hôpital des Enfants-Toulouse, Toulouse (J.P.S.), Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Lyon (M.N., M.R.), and Hôpital Necker-Enfants Malades, Paris (V.C.-D.) - all in France; the University of Alberta-Stollery Children's Hospital, Edmonton, Canada (P.K.); Vanderbilt University Medical Center, Nashville (J.P.); Cincinnati Children's Hospital Medical Center, Cincinnati (H.S.); Benioff Children's Hospital Oakland, Oakland (P.H.), and BridgeBio Pharma, San Francisco (D.H., E.M., R.W., Y.B., D.R.) - both in California; and Johns Hopkins University, Baltimore (J.H.-F.).

Publikováno v: The New England journal of medicine [N Engl J Med] 2024 Nov 18. Date of Electronic Publication: 2024 Nov 18.

Standardised practices in the networked management of congenital hyperinsulinism: a UK national collaborative consensus.

Autor: Shaikh MG; Department of Paediatric Endocrinology, Royal Hospital for Children, Glasgow, United Kingdom., Lucas-Herald AK; Department of Paediatric Endocrinology, Royal Hospital for Children, Glasgow, United Kingdom., Dastamani A; Department of Paediatric Endocrinology and Diabetes, Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom., Salomon Estebanez M; Department of Paediatric Endocrinology, Royal Manchester Children's Hospital, Manchester, United Kingdom., Senniappan S; Department of Paediatric Endocrinology, Alder Hey Children's Hospital, Liverpool, United Kingdom., Abid N; Department of Paediatric Endocrinology, Royal Belfast Hospital for Sick Children, Belfast, United Kingdom., Ahmad S; Department of Paediatric Endocrinology, Royal Manchester Children's Hospital, Manchester, United Kingdom., Alexander S; Department of Paediatric Endocrinology and Diabetes, Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom., Avatapalle B; Department of Paediatric Endocrinology and Diabetes, University Hospital of Wales, Cardiff, United Kingdom., Awan N; Department of Paediatric Endocrinology, Royal Manchester Children's Hospital, Manchester, United Kingdom., Blair H; Department of Dietetics, The Royal Infirmary of Edinburgh, Edinburgh, United Kingdom., Boyle R; Department of Paediatric Endocrinology, Royal Hospital for Children, Glasgow, United Kingdom., Chesover A; Department of Paediatric Endocrinology and Diabetes, Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom., Cochrane B; Department of Paediatric Endocrinology, Royal Hospital for Children, Glasgow, United Kingdom., Craigie R; Department of Paediatric Surgery, Royal Manchester Children's Hospital, Manchester, United Kingdom., Cunjamalay A; Department of Paediatric Endocrinology and Diabetes, Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom., Dearman S; The Children's Hyperinsulinism Charity, Accrington, United Kingdom., De Coppi P; SNAPS, Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom.; NIHR BRC UCL Institute of Child Health, London, United Kingdom., Erlandson-Parry K; Department of Paediatric Endocrinology, Alder Hey Children's Hospital, Liverpool, United Kingdom., Flanagan SE; Department of Clinical and Biomedical Science, University of Exeter, Exeter, United Kingdom., Gilbert C; Department of Paediatric Endocrinology and Diabetes, Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom., Gilligan N; Department of Paediatric Endocrinology, Royal Manchester Children's Hospital, Manchester, United Kingdom., Hall C; Department of Paediatric Endocrinology, Royal Manchester Children's Hospital, Manchester, United Kingdom., Houghton J; Exeter Genomics Laboratory, Royal Devon University Healthcare NHS Foundation Trust, Exeter, United Kingdom., Kapoor R; Department of Paediatric Endocrinology, Faculty of Medicine and Life Sciences, King's College London, King's College Hospital NHS Foundation Trust, London, United Kingdom., McDevitt H; Department of Paediatric Endocrinology, Royal Hospital for Children, Glasgow, United Kingdom., Mohamed Z; Department of Paediatric Endocrinology, Birmingham Children's Hospital, Birmingham, United Kingdom., Morgan K; Department of Paediatric Endocrinology and Diabetes, Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom., Nicholson J; Paediatric Psychosocial Service, Royal Manchester Children's Hospital, Manchester, United Kingdom., Nikiforovski A; Department of Paediatric Endocrinology, Royal Manchester Children's Hospital, Manchester, United Kingdom., O'Shea E; Department of Paediatric Endocrinology, Royal Manchester Children's Hospital, Manchester, United Kingdom., Shah P; Department of Paediatric Endocrinology, Barts Health NHS Trust, Royal London Children's Hospital, London, United Kingdom., Wilson K; Department of Paediatric Endocrinology, Royal Hospital for Children, Glasgow, United Kingdom., Worth C; Department of Paediatric Endocrinology, Royal Manchester Children's Hospital, Manchester, United Kingdom., Worthington S; Department of Paediatric Endocrinology, Royal Manchester Children's Hospital, Manchester, United Kingdom., Banerjee I; Department of Paediatric Endocrinology, Royal Manchester Children's Hospital, Manchester, United Kingdom.

Publikováno v: Frontiers in endocrinology [Front Endocrinol (Lausanne)] 2023 Oct 30; Vol. 14, pp. 1231043. Date of Electronic Publication: 2023 Oct 30 (Print Publication: 2023).

Genomic Polymorphism, Recombination, and Linkage Disequilibrium in Human Major Histocompatibility Complex-Encoded Antigen-Processing Genes

Autor: van Endert, P. M., Lopez, M. T., Patel, S. D., Monaco, J. J., McDevitt, H. O.

Publikováno v: Proceedings of the National Academy of Sciences of the United States of America, 1992 Dec 01. 89(23), 11594-11597.

Externí odkaz: https://www.jstor.org/stable/2360756

Longitudinal changes in bone health as assessed by the speed of sound in very low birth weight preterm infants

Autor: Tomlinson, C., McDevitt, H., Ahmed, S.F., White, M.P.

Publikováno v: In The Journal of Pediatrics 2006 148(4):450-455

THE NATURE OF AUTOIMMUNE-DISEASE - GENERAL DISCUSSION

Autor: MCDEVITT, H, COOKE, A, MITCHISON, N, ROSE, N, NEILSON, E, ROITT, I, NEWSOMDAVIES, J, NOSSAL, G, MACKAY, I, Waldmann, H, LACHMANN, P, PETERS, D, ROSEN, F, UNANUE, E, CARSON, D, WEETMAN, A

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=dedup_wf_001::ea1f435256161aac4d4dde4bcab4a38f
https://ora.ox.ac.uk/objects/uuid:e0eb3ee2-1319-4ff7-a17d-9b6bdfd86412