Safety and Efficacy of Nipocalimab in Patients With Generalized Myasthenia Gravis: Results From the Randomized Phase 2 Vivacity-MG Study

Autor: Antozzi C; From the Neurological Institute Foundation C. Besta (C.A.), Milan, Italy; Duke University School of Medicine (J. Guptill), Durham, NC; Argenx US Inc. (J. Guptill), Boston, MA; University of Toronto (V.B.), ON, Canada; Universitat Autonoma de Barcelona, (J. Gamez), Spain; Medical Faculty (S.G.M.), Heinrich-Heine-University, Düsseldorf, Germany; Yale University School of Medicine (R.J.N.), New Haven, CT; University of Colorado School of Medicine (D.Q.), Aurora; Hospital Universitari i Politécnic La Fe (T.S.), Universitat de Valencia, Spain; Pharvaris, Inc. (M.-H.J.), Boston, MA; Janssen Research & Development, LLC, (J.J., K.K., S.R.,H.S., L.L., Y.Z.), Titusville, NJ; Marinus Pharmaceuticals, Inc. Radnor, PA; Fulcrum Therapeutics (S.A.), Cambridge, MA., Guptill J; From the Neurological Institute Foundation C. Besta (C.A.), Milan, Italy; Duke University School of Medicine (J. Guptill), Durham, NC; Argenx US Inc. (J. Guptill), Boston, MA; University of Toronto (V.B.), ON, Canada; Universitat Autonoma de Barcelona, (J. Gamez), Spain; Medical Faculty (S.G.M.), Heinrich-Heine-University, Düsseldorf, Germany; Yale University School of Medicine (R.J.N.), New Haven, CT; University of Colorado School of Medicine (D.Q.), Aurora; Hospital Universitari i Politécnic La Fe (T.S.), Universitat de Valencia, Spain; Pharvaris, Inc. (M.-H.J.), Boston, MA; Janssen Research & Development, LLC, (J.J., K.K., S.R.,H.S., L.L., Y.Z.), Titusville, NJ; Marinus Pharmaceuticals, Inc. Radnor, PA; Fulcrum Therapeutics (S.A.), Cambridge, MA., Bril V; From the Neurological Institute Foundation C. Besta (C.A.), Milan, Italy; Duke University School of Medicine (J. Guptill), Durham, NC; Argenx US Inc. (J. Guptill), Boston, MA; University of Toronto (V.B.), ON, Canada; Universitat Autonoma de Barcelona, (J. Gamez), Spain; Medical Faculty (S.G.M.), Heinrich-Heine-University, Düsseldorf, Germany; Yale University School of Medicine (R.J.N.), New Haven, CT; University of Colorado School of Medicine (D.Q.), Aurora; Hospital Universitari i Politécnic La Fe (T.S.), Universitat de Valencia, Spain; Pharvaris, Inc. (M.-H.J.), Boston, MA; Janssen Research & Development, LLC, (J.J., K.K., S.R.,H.S., L.L., Y.Z.), Titusville, NJ; Marinus Pharmaceuticals, Inc. Radnor, PA; Fulcrum Therapeutics (S.A.), Cambridge, MA., Gamez J; From the Neurological Institute Foundation C. Besta (C.A.), Milan, Italy; Duke University School of Medicine (J. Guptill), Durham, NC; Argenx US Inc. (J. Guptill), Boston, MA; University of Toronto (V.B.), ON, Canada; Universitat Autonoma de Barcelona, (J. Gamez), Spain; Medical Faculty (S.G.M.), Heinrich-Heine-University, Düsseldorf, Germany; Yale University School of Medicine (R.J.N.), New Haven, CT; University of Colorado School of Medicine (D.Q.), Aurora; Hospital Universitari i Politécnic La Fe (T.S.), Universitat de Valencia, Spain; Pharvaris, Inc. (M.-H.J.), Boston, MA; Janssen Research & Development, LLC, (J.J., K.K., S.R.,H.S., L.L., Y.Z.), Titusville, NJ; Marinus Pharmaceuticals, Inc. Radnor, PA; Fulcrum Therapeutics (S.A.), Cambridge, MA., Meuth SG; From the Neurological Institute Foundation C. Besta (C.A.), Milan, Italy; Duke University School of Medicine (J. Guptill), Durham, NC; Argenx US Inc. (J. Guptill), Boston, MA; University of Toronto (V.B.), ON, Canada; Universitat Autonoma de Barcelona, (J. Gamez), Spain; Medical Faculty (S.G.M.), Heinrich-Heine-University, Düsseldorf, Germany; Yale University School of Medicine (R.J.N.), New Haven, CT; University of Colorado School of Medicine (D.Q.), Aurora; Hospital Universitari i Politécnic La Fe (T.S.), Universitat de Valencia, Spain; Pharvaris, Inc. (M.-H.J.), Boston, MA; Janssen Research & Development, LLC, (J.J., K.K., S.R.,H.S., L.L., Y.Z.), Titusville, NJ; Marinus Pharmaceuticals, Inc. Radnor, PA; Fulcrum Therapeutics (S.A.), Cambridge, MA., Nowak RJ; From the Neurological Institute Foundation C. Besta (C.A.), Milan, Italy; Duke University School of Medicine (J. Guptill), Durham, NC; Argenx US Inc. (J. Guptill), Boston, MA; University of Toronto (V.B.), ON, Canada; Universitat Autonoma de Barcelona, (J. Gamez), Spain; Medical Faculty (S.G.M.), Heinrich-Heine-University, Düsseldorf, Germany; Yale University School of Medicine (R.J.N.), New Haven, CT; University of Colorado School of Medicine (D.Q.), Aurora; Hospital Universitari i Politécnic La Fe (T.S.), Universitat de Valencia, Spain; Pharvaris, Inc. (M.-H.J.), Boston, MA; Janssen Research & Development, LLC, (J.J., K.K., S.R.,H.S., L.L., Y.Z.), Titusville, NJ; Marinus Pharmaceuticals, Inc. Radnor, PA; Fulcrum Therapeutics (S.A.), Cambridge, MA., Quan D; From the Neurological Institute Foundation C. Besta (C.A.), Milan, Italy; Duke University School of Medicine (J. Guptill), Durham, NC; Argenx US Inc. (J. Guptill), Boston, MA; University of Toronto (V.B.), ON, Canada; Universitat Autonoma de Barcelona, (J. Gamez), Spain; Medical Faculty (S.G.M.), Heinrich-Heine-University, Düsseldorf, Germany; Yale University School of Medicine (R.J.N.), New Haven, CT; University of Colorado School of Medicine (D.Q.), Aurora; Hospital Universitari i Politécnic La Fe (T.S.), Universitat de Valencia, Spain; Pharvaris, Inc. (M.-H.J.), Boston, MA; Janssen Research & Development, LLC, (J.J., K.K., S.R.,H.S., L.L., Y.Z.), Titusville, NJ; Marinus Pharmaceuticals, Inc. Radnor, PA; Fulcrum Therapeutics (S.A.), Cambridge, MA., Sevilla T; From the Neurological Institute Foundation C. Besta (C.A.), Milan, Italy; Duke University School of Medicine (J. Guptill), Durham, NC; Argenx US Inc. (J. Guptill), Boston, MA; University of Toronto (V.B.), ON, Canada; Universitat Autonoma de Barcelona, (J. Gamez), Spain; Medical Faculty (S.G.M.), Heinrich-Heine-University, Düsseldorf, Germany; Yale University School of Medicine (R.J.N.), New Haven, CT; University of Colorado School of Medicine (D.Q.), Aurora; Hospital Universitari i Politécnic La Fe (T.S.), Universitat de Valencia, Spain; Pharvaris, Inc. (M.-H.J.), Boston, MA; Janssen Research & Development, LLC, (J.J., K.K., S.R.,H.S., L.L., Y.Z.), Titusville, NJ; Marinus Pharmaceuticals, Inc. Radnor, PA; Fulcrum Therapeutics (S.A.), Cambridge, MA., Jouvin MH; From the Neurological Institute Foundation C. Besta (C.A.), Milan, Italy; Duke University School of Medicine (J. Guptill), Durham, NC; Argenx US Inc. (J. Guptill), Boston, MA; University of Toronto (V.B.), ON, Canada; Universitat Autonoma de Barcelona, (J. Gamez), Spain; Medical Faculty (S.G.M.), Heinrich-Heine-University, Düsseldorf, Germany; Yale University School of Medicine (R.J.N.), New Haven, CT; University of Colorado School of Medicine (D.Q.), Aurora; Hospital Universitari i Politécnic La Fe (T.S.), Universitat de Valencia, Spain; Pharvaris, Inc. (M.-H.J.), Boston, MA; Janssen Research & Development, LLC, (J.J., K.K., S.R.,H.S., L.L., Y.Z.), Titusville, NJ; Marinus Pharmaceuticals, Inc. Radnor, PA; Fulcrum Therapeutics (S.A.), Cambridge, MA., Jin J; From the Neurological Institute Foundation C. Besta (C.A.), Milan, Italy; Duke University School of Medicine (J. Guptill), Durham, NC; Argenx US Inc. (J. Guptill), Boston, MA; University of Toronto (V.B.), ON, Canada; Universitat Autonoma de Barcelona, (J. Gamez), Spain; Medical Faculty (S.G.M.), Heinrich-Heine-University, Düsseldorf, Germany; Yale University School of Medicine (R.J.N.), New Haven, CT; University of Colorado School of Medicine (D.Q.), Aurora; Hospital Universitari i Politécnic La Fe (T.S.), Universitat de Valencia, Spain; Pharvaris, Inc. (M.-H.J.), Boston, MA; Janssen Research & Development, LLC, (J.J., K.K., S.R.,H.S., L.L., Y.Z.), Titusville, NJ; Marinus Pharmaceuticals, Inc. Radnor, PA; Fulcrum Therapeutics (S.A.), Cambridge, MA., Karcher K; From the Neurological Institute Foundation C. Besta (C.A.), Milan, Italy; Duke University School of Medicine (J. Guptill), Durham, NC; Argenx US Inc. (J. Guptill), Boston, MA; University of Toronto (V.B.), ON, Canada; Universitat Autonoma de Barcelona, (J. Gamez), Spain; Medical Faculty (S.G.M.), Heinrich-Heine-University, Düsseldorf, Germany; Yale University School of Medicine (R.J.N.), New Haven, CT; University of Colorado School of Medicine (D.Q.), Aurora; Hospital Universitari i Politécnic La Fe (T.S.), Universitat de Valencia, Spain; Pharvaris, Inc. (M.-H.J.), Boston, MA; Janssen Research & Development, LLC, (J.J., K.K., S.R.,H.S., L.L., Y.Z.), Titusville, NJ; Marinus Pharmaceuticals, Inc. Radnor, PA; Fulcrum Therapeutics (S.A.), Cambridge, MA., Ramchandren S; From the Neurological Institute Foundation C. Besta (C.A.), Milan, Italy; Duke University School of Medicine (J. Guptill), Durham, NC; Argenx US Inc. (J. Guptill), Boston, MA; University of Toronto (V.B.), ON, Canada; Universitat Autonoma de Barcelona, (J. Gamez), Spain; Medical Faculty (S.G.M.), Heinrich-Heine-University, Düsseldorf, Germany; Yale University School of Medicine (R.J.N.), New Haven, CT; University of Colorado School of Medicine (D.Q.), Aurora; Hospital Universitari i Politécnic La Fe (T.S.), Universitat de Valencia, Spain; Pharvaris, Inc. (M.-H.J.), Boston, MA; Janssen Research & Development, LLC, (J.J., K.K., S.R.,H.S., L.L., Y.Z.), Titusville, NJ; Marinus Pharmaceuticals, Inc. Radnor, PA; Fulcrum Therapeutics (S.A.), Cambridge, MA., Sun H; From the Neurological Institute Foundation C. Besta (C.A.), Milan, Italy; Duke University School of Medicine (J. Guptill), Durham, NC; Argenx US Inc. (J. Guptill), Boston, MA; University of Toronto (V.B.), ON, Canada; Universitat Autonoma de Barcelona, (J. Gamez), Spain; Medical Faculty (S.G.M.), Heinrich-Heine-University, Düsseldorf, Germany; Yale University School of Medicine (R.J.N.), New Haven, CT; University of Colorado School of Medicine (D.Q.), Aurora; Hospital Universitari i Politécnic La Fe (T.S.), Universitat de Valencia, Spain; Pharvaris, Inc. (M.-H.J.), Boston, MA; Janssen Research & Development, LLC, (J.J., K.K., S.R.,H.S., L.L., Y.Z.), Titusville, NJ; Marinus Pharmaceuticals, Inc. Radnor, PA; Fulcrum Therapeutics (S.A.), Cambridge, MA., Ling L; From the Neurological Institute Foundation C. Besta (C.A.), Milan, Italy; Duke University School of Medicine (J. Guptill), Durham, NC; Argenx US Inc. (J. Guptill), Boston, MA; University of Toronto (V.B.), ON, Canada; Universitat Autonoma de Barcelona, (J. Gamez), Spain; Medical Faculty (S.G.M.), Heinrich-Heine-University, Düsseldorf, Germany; Yale University School of Medicine (R.J.N.), New Haven, CT; University of Colorado School of Medicine (D.Q.), Aurora; Hospital Universitari i Politécnic La Fe (T.S.), Universitat de Valencia, Spain; Pharvaris, Inc. (M.-H.J.), Boston, MA; Janssen Research & Development, LLC, (J.J., K.K., S.R.,H.S., L.L., Y.Z.), Titusville, NJ; Marinus Pharmaceuticals, Inc. Radnor, PA; Fulcrum Therapeutics (S.A.), Cambridge, MA., Zhu Y; From the Neurological Institute Foundation C. Besta (C.A.), Milan, Italy; Duke University School of Medicine (J. Guptill), Durham, NC; Argenx US Inc. (J. Guptill), Boston, MA; University of Toronto (V.B.), ON, Canada; Universitat Autonoma de Barcelona, (J. Gamez), Spain; Medical Faculty (S.G.M.), Heinrich-Heine-University, Düsseldorf, Germany; Yale University School of Medicine (R.J.N.), New Haven, CT; University of Colorado School of Medicine (D.Q.), Aurora; Hospital Universitari i Politécnic La Fe (T.S.), Universitat de Valencia, Spain; Pharvaris, Inc. (M.-H.J.), Boston, MA; Janssen Research & Development, LLC, (J.J., K.K., S.R.,H.S., L.L., Y.Z.), Titusville, NJ; Marinus Pharmaceuticals, Inc. Radnor, PA; Fulcrum Therapeutics (S.A.), Cambridge, MA., Arroyo S; From the Neurological Institute Foundation C. Besta (C.A.), Milan, Italy; Duke University School of Medicine (J. Guptill), Durham, NC; Argenx US Inc. (J. Guptill), Boston, MA; University of Toronto (V.B.), ON, Canada; Universitat Autonoma de Barcelona, (J. Gamez), Spain; Medical Faculty (S.G.M.), Heinrich-Heine-University, Düsseldorf, Germany; Yale University School of Medicine (R.J.N.), New Haven, CT; University of Colorado School of Medicine (D.Q.), Aurora; Hospital Universitari i Politécnic La Fe (T.S.), Universitat de Valencia, Spain; Pharvaris, Inc. (M.-H.J.), Boston, MA; Janssen Research & Development, LLC, (J.J., K.K., S.R.,H.S., L.L., Y.Z.), Titusville, NJ; Marinus Pharmaceuticals, Inc. Radnor, PA; Fulcrum Therapeutics (S.A.), Cambridge, MA.

Publikováno v: Neurology [Neurology] 2024 Jan 23; Vol. 102 (2), pp. e207937. Date of Electronic Publication: 2023 Dec 21.

A novel risk stratification approach and molecular subgroup characterization based on coagulation related genes in colon adenocarcinoma.

Autor: Wu, Xiangxin¹ (AUTHOR), Zhu, Lichong² (AUTHOR), Sun, Xizhe³ (AUTHOR), Xia, Mingyu⁴ (AUTHOR), Zhao, Shihui⁴ (AUTHOR), Zhang, Bomiao⁴ (AUTHOR), Xia, Tianyi⁴ (AUTHOR) xiatianyi@hrbmu.edu.cn

Publikováno v: Cancer Cell International. 9/9/2024, Vol. 24 Issue 1, p1-16. 16p.

Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency.

Autor: Grace RF; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston (R.F.G.), and Agios Pharmaceuticals, Cambridge (A.J.B., S.B., L.H., C.K., P.H., M.-H.J., C.B.) - all in Massachusetts; Hôpital Saint Vincent de Paul, Lille (C.R.), and Unité des Maladies Génétiques du Globule Rouge, Centre Hospitalier Universitaire Henri Mondor, Créteil (F.G.) - both in France; Hammersmith Hospital, Imperial College Healthcare NHS Trust, London (D.M.L.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.); Central Pennsylvania Clinic, Belleville (D.H.M.), and Children's Hospital of Philadelphia and Perelman School of Medicine of the University of Pennsylvania, Philadelphia (J.L.K.); Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (E.J.B.); University of Utah, Salt Lake City (H.Y.); Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit (Y.R.); University of Toronto, Toronto (K.H.M.K.); Weill Cornell Medical College, New York (S.S.); Bruce A. Silver Clinical Science and Development, Dunkirk, MD (B.S.); and Stanford University School of Medicine, Palo Alto, CA (B.G.)., Rose C; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston (R.F.G.), and Agios Pharmaceuticals, Cambridge (A.J.B., S.B., L.H., C.K., P.H., M.-H.J., C.B.) - all in Massachusetts; Hôpital Saint Vincent de Paul, Lille (C.R.), and Unité des Maladies Génétiques du Globule Rouge, Centre Hospitalier Universitaire Henri Mondor, Créteil (F.G.) - both in France; Hammersmith Hospital, Imperial College Healthcare NHS Trust, London (D.M.L.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.); Central Pennsylvania Clinic, Belleville (D.H.M.), and Children's Hospital of Philadelphia and Perelman School of Medicine of the University of Pennsylvania, Philadelphia (J.L.K.); Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (E.J.B.); University of Utah, Salt Lake City (H.Y.); Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit (Y.R.); University of Toronto, Toronto (K.H.M.K.); Weill Cornell Medical College, New York (S.S.); Bruce A. Silver Clinical Science and Development, Dunkirk, MD (B.S.); and Stanford University School of Medicine, Palo Alto, CA (B.G.)., Layton DM; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston (R.F.G.), and Agios Pharmaceuticals, Cambridge (A.J.B., S.B., L.H., C.K., P.H., M.-H.J., C.B.) - all in Massachusetts; Hôpital Saint Vincent de Paul, Lille (C.R.), and Unité des Maladies Génétiques du Globule Rouge, Centre Hospitalier Universitaire Henri Mondor, Créteil (F.G.) - both in France; Hammersmith Hospital, Imperial College Healthcare NHS Trust, London (D.M.L.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.); Central Pennsylvania Clinic, Belleville (D.H.M.), and Children's Hospital of Philadelphia and Perelman School of Medicine of the University of Pennsylvania, Philadelphia (J.L.K.); Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (E.J.B.); University of Utah, Salt Lake City (H.Y.); Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit (Y.R.); University of Toronto, Toronto (K.H.M.K.); Weill Cornell Medical College, New York (S.S.); Bruce A. Silver Clinical Science and Development, Dunkirk, MD (B.S.); and Stanford University School of Medicine, Palo Alto, CA (B.G.)., Galactéros F; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston (R.F.G.), and Agios Pharmaceuticals, Cambridge (A.J.B., S.B., L.H., C.K., P.H., M.-H.J., C.B.) - all in Massachusetts; Hôpital Saint Vincent de Paul, Lille (C.R.), and Unité des Maladies Génétiques du Globule Rouge, Centre Hospitalier Universitaire Henri Mondor, Créteil (F.G.) - both in France; Hammersmith Hospital, Imperial College Healthcare NHS Trust, London (D.M.L.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.); Central Pennsylvania Clinic, Belleville (D.H.M.), and Children's Hospital of Philadelphia and Perelman School of Medicine of the University of Pennsylvania, Philadelphia (J.L.K.); Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (E.J.B.); University of Utah, Salt Lake City (H.Y.); Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit (Y.R.); University of Toronto, Toronto (K.H.M.K.); Weill Cornell Medical College, New York (S.S.); Bruce A. Silver Clinical Science and Development, Dunkirk, MD (B.S.); and Stanford University School of Medicine, Palo Alto, CA (B.G.)., Barcellini W; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston (R.F.G.), and Agios Pharmaceuticals, Cambridge (A.J.B., S.B., L.H., C.K., P.H., M.-H.J., C.B.) - all in Massachusetts; Hôpital Saint Vincent de Paul, Lille (C.R.), and Unité des Maladies Génétiques du Globule Rouge, Centre Hospitalier Universitaire Henri Mondor, Créteil (F.G.) - both in France; Hammersmith Hospital, Imperial College Healthcare NHS Trust, London (D.M.L.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.); Central Pennsylvania Clinic, Belleville (D.H.M.), and Children's Hospital of Philadelphia and Perelman School of Medicine of the University of Pennsylvania, Philadelphia (J.L.K.); Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (E.J.B.); University of Utah, Salt Lake City (H.Y.); Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit (Y.R.); University of Toronto, Toronto (K.H.M.K.); Weill Cornell Medical College, New York (S.S.); Bruce A. Silver Clinical Science and Development, Dunkirk, MD (B.S.); and Stanford University School of Medicine, Palo Alto, CA (B.G.)., Morton DH; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston (R.F.G.), and Agios Pharmaceuticals, Cambridge (A.J.B., S.B., L.H., C.K., P.H., M.-H.J., C.B.) - all in Massachusetts; Hôpital Saint Vincent de Paul, Lille (C.R.), and Unité des Maladies Génétiques du Globule Rouge, Centre Hospitalier Universitaire Henri Mondor, Créteil (F.G.) - both in France; Hammersmith Hospital, Imperial College Healthcare NHS Trust, London (D.M.L.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.); Central Pennsylvania Clinic, Belleville (D.H.M.), and Children's Hospital of Philadelphia and Perelman School of Medicine of the University of Pennsylvania, Philadelphia (J.L.K.); Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (E.J.B.); University of Utah, Salt Lake City (H.Y.); Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit (Y.R.); University of Toronto, Toronto (K.H.M.K.); Weill Cornell Medical College, New York (S.S.); Bruce A. Silver Clinical Science and Development, Dunkirk, MD (B.S.); and Stanford University School of Medicine, Palo Alto, CA (B.G.)., van Beers EJ; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston (R.F.G.), and Agios Pharmaceuticals, Cambridge (A.J.B., S.B., L.H., C.K., P.H., M.-H.J., C.B.) - all in Massachusetts; Hôpital Saint Vincent de Paul, Lille (C.R.), and Unité des Maladies Génétiques du Globule Rouge, Centre Hospitalier Universitaire Henri Mondor, Créteil (F.G.) - both in France; Hammersmith Hospital, Imperial College Healthcare NHS Trust, London (D.M.L.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.); Central Pennsylvania Clinic, Belleville (D.H.M.), and Children's Hospital of Philadelphia and Perelman School of Medicine of the University of Pennsylvania, Philadelphia (J.L.K.); Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (E.J.B.); University of Utah, Salt Lake City (H.Y.); Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit (Y.R.); University of Toronto, Toronto (K.H.M.K.); Weill Cornell Medical College, New York (S.S.); Bruce A. Silver Clinical Science and Development, Dunkirk, MD (B.S.); and Stanford University School of Medicine, Palo Alto, CA (B.G.)., Yaish H; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston (R.F.G.), and Agios Pharmaceuticals, Cambridge (A.J.B., S.B., L.H., C.K., P.H., M.-H.J., C.B.) - all in Massachusetts; Hôpital Saint Vincent de Paul, Lille (C.R.), and Unité des Maladies Génétiques du Globule Rouge, Centre Hospitalier Universitaire Henri Mondor, Créteil (F.G.) - both in France; Hammersmith Hospital, Imperial College Healthcare NHS Trust, London (D.M.L.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.); Central Pennsylvania Clinic, Belleville (D.H.M.), and Children's Hospital of Philadelphia and Perelman School of Medicine of the University of Pennsylvania, Philadelphia (J.L.K.); Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (E.J.B.); University of Utah, Salt Lake City (H.Y.); Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit (Y.R.); University of Toronto, Toronto (K.H.M.K.); Weill Cornell Medical College, New York (S.S.); Bruce A. Silver Clinical Science and Development, Dunkirk, MD (B.S.); and Stanford University School of Medicine, Palo Alto, CA (B.G.)., Ravindranath Y; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston (R.F.G.), and Agios Pharmaceuticals, Cambridge (A.J.B., S.B., L.H., C.K., P.H., M.-H.J., C.B.) - all in Massachusetts; Hôpital Saint Vincent de Paul, Lille (C.R.), and Unité des Maladies Génétiques du Globule Rouge, Centre Hospitalier Universitaire Henri Mondor, Créteil (F.G.) - both in France; Hammersmith Hospital, Imperial College Healthcare NHS Trust, London (D.M.L.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.); Central Pennsylvania Clinic, Belleville (D.H.M.), and Children's Hospital of Philadelphia and Perelman School of Medicine of the University of Pennsylvania, Philadelphia (J.L.K.); Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (E.J.B.); University of Utah, Salt Lake City (H.Y.); Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit (Y.R.); University of Toronto, Toronto (K.H.M.K.); Weill Cornell Medical College, New York (S.S.); Bruce A. Silver Clinical Science and Development, Dunkirk, MD (B.S.); and Stanford University School of Medicine, Palo Alto, CA (B.G.)., Kuo KHM; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston (R.F.G.), and Agios Pharmaceuticals, Cambridge (A.J.B., S.B., L.H., C.K., P.H., M.-H.J., C.B.) - all in Massachusetts; Hôpital Saint Vincent de Paul, Lille (C.R.), and Unité des Maladies Génétiques du Globule Rouge, Centre Hospitalier Universitaire Henri Mondor, Créteil (F.G.) - both in France; Hammersmith Hospital, Imperial College Healthcare NHS Trust, London (D.M.L.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.); Central Pennsylvania Clinic, Belleville (D.H.M.), and Children's Hospital of Philadelphia and Perelman School of Medicine of the University of Pennsylvania, Philadelphia (J.L.K.); Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (E.J.B.); University of Utah, Salt Lake City (H.Y.); Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit (Y.R.); University of Toronto, Toronto (K.H.M.K.); Weill Cornell Medical College, New York (S.S.); Bruce A. Silver Clinical Science and Development, Dunkirk, MD (B.S.); and Stanford University School of Medicine, Palo Alto, CA (B.G.)., Sheth S; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston (R.F.G.), and Agios Pharmaceuticals, Cambridge (A.J.B., S.B., L.H., C.K., P.H., M.-H.J., C.B.) - all in Massachusetts; Hôpital Saint Vincent de Paul, Lille (C.R.), and Unité des Maladies Génétiques du Globule Rouge, Centre Hospitalier Universitaire Henri Mondor, Créteil (F.G.) - both in France; Hammersmith Hospital, Imperial College Healthcare NHS Trust, London (D.M.L.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.); Central Pennsylvania Clinic, Belleville (D.H.M.), and Children's Hospital of Philadelphia and Perelman School of Medicine of the University of Pennsylvania, Philadelphia (J.L.K.); Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (E.J.B.); University of Utah, Salt Lake City (H.Y.); Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit (Y.R.); University of Toronto, Toronto (K.H.M.K.); Weill Cornell Medical College, New York (S.S.); Bruce A. Silver Clinical Science and Development, Dunkirk, MD (B.S.); and Stanford University School of Medicine, Palo Alto, CA (B.G.)., Kwiatkowski JL; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston (R.F.G.), and Agios Pharmaceuticals, Cambridge (A.J.B., S.B., L.H., C.K., P.H., M.-H.J., C.B.) - all in Massachusetts; Hôpital Saint Vincent de Paul, Lille (C.R.), and Unité des Maladies Génétiques du Globule Rouge, Centre Hospitalier Universitaire Henri Mondor, Créteil (F.G.) - both in France; Hammersmith Hospital, Imperial College Healthcare NHS Trust, London (D.M.L.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.); Central Pennsylvania Clinic, Belleville (D.H.M.), and Children's Hospital of Philadelphia and Perelman School of Medicine of the University of Pennsylvania, Philadelphia (J.L.K.); Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (E.J.B.); University of Utah, Salt Lake City (H.Y.); Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit (Y.R.); University of Toronto, Toronto (K.H.M.K.); Weill Cornell Medical College, New York (S.S.); Bruce A. Silver Clinical Science and Development, Dunkirk, MD (B.S.); and Stanford University School of Medicine, Palo Alto, CA (B.G.)., Barbier AJ; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston (R.F.G.), and Agios Pharmaceuticals, Cambridge (A.J.B., S.B., L.H., C.K., P.H., M.-H.J., C.B.) - all in Massachusetts; Hôpital Saint Vincent de Paul, Lille (C.R.), and Unité des Maladies Génétiques du Globule Rouge, Centre Hospitalier Universitaire Henri Mondor, Créteil (F.G.) - both in France; Hammersmith Hospital, Imperial College Healthcare NHS Trust, London (D.M.L.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.); Central Pennsylvania Clinic, Belleville (D.H.M.), and Children's Hospital of Philadelphia and Perelman School of Medicine of the University of Pennsylvania, Philadelphia (J.L.K.); Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (E.J.B.); University of Utah, Salt Lake City (H.Y.); Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit (Y.R.); University of Toronto, Toronto (K.H.M.K.); Weill Cornell Medical College, New York (S.S.); Bruce A. Silver Clinical Science and Development, Dunkirk, MD (B.S.); and Stanford University School of Medicine, Palo Alto, CA (B.G.)., Bodie S; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston (R.F.G.), and Agios Pharmaceuticals, Cambridge (A.J.B., S.B., L.H., C.K., P.H., M.-H.J., C.B.) - all in Massachusetts; Hôpital Saint Vincent de Paul, Lille (C.R.), and Unité des Maladies Génétiques du Globule Rouge, Centre Hospitalier Universitaire Henri Mondor, Créteil (F.G.) - both in France; Hammersmith Hospital, Imperial College Healthcare NHS Trust, London (D.M.L.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.); Central Pennsylvania Clinic, Belleville (D.H.M.), and Children's Hospital of Philadelphia and Perelman School of Medicine of the University of Pennsylvania, Philadelphia (J.L.K.); Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (E.J.B.); University of Utah, Salt Lake City (H.Y.); Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit (Y.R.); University of Toronto, Toronto (K.H.M.K.); Weill Cornell Medical College, New York (S.S.); Bruce A. Silver Clinical Science and Development, Dunkirk, MD (B.S.); and Stanford University School of Medicine, Palo Alto, CA (B.G.)., Silver B; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston (R.F.G.), and Agios Pharmaceuticals, Cambridge (A.J.B., S.B., L.H., C.K., P.H., M.-H.J., C.B.) - all in Massachusetts; Hôpital Saint Vincent de Paul, Lille (C.R.), and Unité des Maladies Génétiques du Globule Rouge, Centre Hospitalier Universitaire Henri Mondor, Créteil (F.G.) - both in France; Hammersmith Hospital, Imperial College Healthcare NHS Trust, London (D.M.L.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.); Central Pennsylvania Clinic, Belleville (D.H.M.), and Children's Hospital of Philadelphia and Perelman School of Medicine of the University of Pennsylvania, Philadelphia (J.L.K.); Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (E.J.B.); University of Utah, Salt Lake City (H.Y.); Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit (Y.R.); University of Toronto, Toronto (K.H.M.K.); Weill Cornell Medical College, New York (S.S.); Bruce A. Silver Clinical Science and Development, Dunkirk, MD (B.S.); and Stanford University School of Medicine, Palo Alto, CA (B.G.)., Hua L; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston (R.F.G.), and Agios Pharmaceuticals, Cambridge (A.J.B., S.B., L.H., C.K., P.H., M.-H.J., C.B.) - all in Massachusetts; Hôpital Saint Vincent de Paul, Lille (C.R.), and Unité des Maladies Génétiques du Globule Rouge, Centre Hospitalier Universitaire Henri Mondor, Créteil (F.G.) - both in France; Hammersmith Hospital, Imperial College Healthcare NHS Trust, London (D.M.L.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.); Central Pennsylvania Clinic, Belleville (D.H.M.), and Children's Hospital of Philadelphia and Perelman School of Medicine of the University of Pennsylvania, Philadelphia (J.L.K.); Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (E.J.B.); University of Utah, Salt Lake City (H.Y.); Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit (Y.R.); University of Toronto, Toronto (K.H.M.K.); Weill Cornell Medical College, New York (S.S.); Bruce A. Silver Clinical Science and Development, Dunkirk, MD (B.S.); and Stanford University School of Medicine, Palo Alto, CA (B.G.)., Kung C; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston (R.F.G.), and Agios Pharmaceuticals, Cambridge (A.J.B., S.B., L.H., C.K., P.H., M.-H.J., C.B.) - all in Massachusetts; Hôpital Saint Vincent de Paul, Lille (C.R.), and Unité des Maladies Génétiques du Globule Rouge, Centre Hospitalier Universitaire Henri Mondor, Créteil (F.G.) - both in France; Hammersmith Hospital, Imperial College Healthcare NHS Trust, London (D.M.L.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.); Central Pennsylvania Clinic, Belleville (D.H.M.), and Children's Hospital of Philadelphia and Perelman School of Medicine of the University of Pennsylvania, Philadelphia (J.L.K.); Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (E.J.B.); University of Utah, Salt Lake City (H.Y.); Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit (Y.R.); University of Toronto, Toronto (K.H.M.K.); Weill Cornell Medical College, New York (S.S.); Bruce A. Silver Clinical Science and Development, Dunkirk, MD (B.S.); and Stanford University School of Medicine, Palo Alto, CA (B.G.)., Hawkins P; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston (R.F.G.), and Agios Pharmaceuticals, Cambridge (A.J.B., S.B., L.H., C.K., P.H., M.-H.J., C.B.) - all in Massachusetts; Hôpital Saint Vincent de Paul, Lille (C.R.), and Unité des Maladies Génétiques du Globule Rouge, Centre Hospitalier Universitaire Henri Mondor, Créteil (F.G.) - both in France; Hammersmith Hospital, Imperial College Healthcare NHS Trust, London (D.M.L.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.); Central Pennsylvania Clinic, Belleville (D.H.M.), and Children's Hospital of Philadelphia and Perelman School of Medicine of the University of Pennsylvania, Philadelphia (J.L.K.); Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (E.J.B.); University of Utah, Salt Lake City (H.Y.); Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit (Y.R.); University of Toronto, Toronto (K.H.M.K.); Weill Cornell Medical College, New York (S.S.); Bruce A. Silver Clinical Science and Development, Dunkirk, MD (B.S.); and Stanford University School of Medicine, Palo Alto, CA (B.G.)., Jouvin MH; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston (R.F.G.), and Agios Pharmaceuticals, Cambridge (A.J.B., S.B., L.H., C.K., P.H., M.-H.J., C.B.) - all in Massachusetts; Hôpital Saint Vincent de Paul, Lille (C.R.), and Unité des Maladies Génétiques du Globule Rouge, Centre Hospitalier Universitaire Henri Mondor, Créteil (F.G.) - both in France; Hammersmith Hospital, Imperial College Healthcare NHS Trust, London (D.M.L.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.); Central Pennsylvania Clinic, Belleville (D.H.M.), and Children's Hospital of Philadelphia and Perelman School of Medicine of the University of Pennsylvania, Philadelphia (J.L.K.); Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (E.J.B.); University of Utah, Salt Lake City (H.Y.); Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit (Y.R.); University of Toronto, Toronto (K.H.M.K.); Weill Cornell Medical College, New York (S.S.); Bruce A. Silver Clinical Science and Development, Dunkirk, MD (B.S.); and Stanford University School of Medicine, Palo Alto, CA (B.G.)., Bowden C; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston (R.F.G.), and Agios Pharmaceuticals, Cambridge (A.J.B., S.B., L.H., C.K., P.H., M.-H.J., C.B.) - all in Massachusetts; Hôpital Saint Vincent de Paul, Lille (C.R.), and Unité des Maladies Génétiques du Globule Rouge, Centre Hospitalier Universitaire Henri Mondor, Créteil (F.G.) - both in France; Hammersmith Hospital, Imperial College Healthcare NHS Trust, London (D.M.L.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.); Central Pennsylvania Clinic, Belleville (D.H.M.), and Children's Hospital of Philadelphia and Perelman School of Medicine of the University of Pennsylvania, Philadelphia (J.L.K.); Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (E.J.B.); University of Utah, Salt Lake City (H.Y.); Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit (Y.R.); University of Toronto, Toronto (K.H.M.K.); Weill Cornell Medical College, New York (S.S.); Bruce A. Silver Clinical Science and Development, Dunkirk, MD (B.S.); and Stanford University School of Medicine, Palo Alto, CA (B.G.)., Glader B; From the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston (R.F.G.), and Agios Pharmaceuticals, Cambridge (A.J.B., S.B., L.H., C.K., P.H., M.-H.J., C.B.) - all in Massachusetts; Hôpital Saint Vincent de Paul, Lille (C.R.), and Unité des Maladies Génétiques du Globule Rouge, Centre Hospitalier Universitaire Henri Mondor, Créteil (F.G.) - both in France; Hammersmith Hospital, Imperial College Healthcare NHS Trust, London (D.M.L.); Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (W.B.); Central Pennsylvania Clinic, Belleville (D.H.M.), and Children's Hospital of Philadelphia and Perelman School of Medicine of the University of Pennsylvania, Philadelphia (J.L.K.); Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands (E.J.B.); University of Utah, Salt Lake City (H.Y.); Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit (Y.R.); University of Toronto, Toronto (K.H.M.K.); Weill Cornell Medical College, New York (S.S.); Bruce A. Silver Clinical Science and Development, Dunkirk, MD (B.S.); and Stanford University School of Medicine, Palo Alto, CA (B.G.).

Publikováno v: The New England journal of medicine [N Engl J Med] 2019 Sep 05; Vol. 381 (10), pp. 933-944.

Once- versus twice-daily dosing of eliglustat in adults with Gaucher disease type 1: The Phase 3, randomized, double-blind EDGE trial.

Autor: Charrow J; Northwestern University Feinberg School of Medicine, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA. Electronic address: jcharrow@northwestern.edu., Fraga C; HDES Hospital, Ponta Delgada, Açores, Portugal., Gu X; Xin Hua Hospital, Shanghai Jiaotong University School of Medicine, Shanghai, China., Ida H; The Jikei University School of Medicine, Tokyo, Japan., Longo N; University of Utah School of Medicine, Salt Lake City, UT, USA., Lukina E; National Research Center for Hematology, Moscow, Russia., Nonino A; Centro de Tratamento de Oncologia e Hematologia (CETTRO), Brasilia, Brazil., Gaemers SJM; Sanofi Genzyme, Cambridge, MA, USA., Jouvin MH; Sanofi Genzyme, Cambridge, MA, USA., Li J; Sanofi Genzyme, Cambridge, MA, USA., Wu Y; Sanofi Genzyme, Cambridge, MA, USA., Xue Y; Sanofi Genzyme, Cambridge, MA, USA., Peterschmitt MJ; Sanofi Genzyme, Cambridge, MA, USA.

Publikováno v: Molecular genetics and metabolism [Mol Genet Metab] 2018 Mar; Vol. 123 (3), pp. 347-356. Date of Electronic Publication: 2018 Jan 04.

Structure and function of FcepsilonRI and of new receptors for immunoglobulin E

Autor: Kinet, Jp, Adamczewski, M, Brini, A, Jouvin, Mh, Kuster, H, Letourneur, O, Paolini, Rossella, Takizawa, F, Zhang, L.

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=od______3686::ce2df4921446972786fde7d2fa5c615e
http://hdl.handle.net/11573/384139