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Effect of the Histone Deacetylase Inhibitor FRM-0334 on Progranulin Levels in Patients With Progranulin Gene Haploinsufficiency: A Randomized Clinical Trial.
Ljubenkov, Peter A; Edwards, Lauren; Iaccarino, Leonardo; La Joie, Renaud; Rojas, Julio C; Koestler, Mary; Harris, Baruch; Boeve, Bradley F; Borroni, Barbara; van Swieten, John C; Grossman, Murray; Pasquier, Florence; Frisoni, Giovanni B; Mummery, Catherine J; Vandenberghe, Rik; Le Ber, Isabelle; Hannequin, Didier; McGinnis, Scott M; Auriacombe, Sophie; Onofrj, Marco; Goodman, Ira J; Riordan, Henry J; Wisniewski, Gary; Hesterman, Jacob; Marek, Ken; Haynes, Beth Ann; Patzke, Holger; Koenig, Gerhard; Hilt, Dana; Moebius, Hans; Boxer, Adam L.
Afiliação
  • Ljubenkov PA; Memory and Aging Center, Department of Neurology, University of California at San Francisco, Weill Institute for Neurosciences, San Francisco.
  • Edwards L; Memory and Aging Center, Department of Neurology, University of California at San Francisco, Weill Institute for Neurosciences, San Francisco.
  • Iaccarino L; Memory and Aging Center, Department of Neurology, University of California at San Francisco, Weill Institute for Neurosciences, San Francisco.
  • La Joie R; Memory and Aging Center, Department of Neurology, University of California at San Francisco, Weill Institute for Neurosciences, San Francisco.
  • Rojas JC; Memory and Aging Center, Department of Neurology, University of California at San Francisco, Weill Institute for Neurosciences, San Francisco.
  • Koestler M; Memory and Aging Center, Department of Neurology, University of California at San Francisco, Weill Institute for Neurosciences, San Francisco.
  • Harris B; ROME Therapeutics, Cambridge, Massachusetts.
  • Boeve BF; Metera Pharmaceuticals, Cambridge, Massachusetts.
  • Borroni B; Mayo Clinic, Rochester, Minnesota.
  • van Swieten JC; Neurology Unit, Centre for Neurodegenerative Disorders, ASST Spedali Civili Brescia, Italy.
  • Grossman M; Department of Clinical and Experimental Sciences, University of Brescia, Italy.
  • Pasquier F; Department of Neurology, Erasmus University Medical Center, Rotterdam, the Netherlands.
  • Frisoni GB; Perelman School of Medicine, University of Pennsylvania, Philadelphia.
  • Mummery CJ; Lille University, Inserm 1172, CHU Lille, CNR-MAJ, DISTALZ, LiCEND, Lille, France.
  • Vandenberghe R; Lab of Alzheimer's Neuroimaging and Epidemiology, IRCCS Istituto Centro San Giovanni di Dio Fatebenefratelli, Brescia, Italy.
  • Le Ber I; Dementia Research Centre, Institute of Neurology, University College London, London, United Kingdom.
  • Hannequin D; Neurology Service University Hospitals Leuven, Leuven, Belgium.
  • McGinnis SM; Laboratory for Cognitive Neurology, Department of Neurosciences, Leuven Brain Institute, Leuven, Belgium.
  • Auriacombe S; Paris Brain Institute, Sorbonne University, Paris, France.
  • Onofrj M; Neurology Department, Reference Centre for Rare or Early Dementias, Paris, France.
  • Goodman IJ; CHU Rouen Normandie, Service de Neurologie, Rouen University Hospital, Rouen, France.
  • Riordan HJ; Department of Neurology, Massachusetts General Hospital, Charlestown.
  • Wisniewski G; Neurology Department, CHU Bordeaux, Bordeaux Hospital, Bordeaux, France.
  • Hesterman J; Department of Neuroscience Imaging, University G d'Annunzio of Chieti-Pescara, Chieti, Italy.
  • Marek K; Compass Research, Bioclinica, Orlando, Florida.
  • Haynes BA; Worldwide Clinical Trials, King of Prussia, Pennsylvania.
  • Patzke H; Invicro, A Konica Minolta Company, Boston, Massachusetts.
  • Koenig G; Institute for Neurodegenerative Disorders, New Haven, Connecticut.
  • Hilt D; Invicro, A Konica Minolta Company, Boston, Massachusetts.
  • Moebius H; Institute for Neurodegenerative Disorders, New Haven, Connecticut.
  • Boxer AL; FORUM Pharmaceuticals Incorporated, Waltham, Massachusetts.
JAMA Netw Open ; 4(9): e2125584, 2021 09 01.
Article em En | MEDLINE | ID: mdl-34559230
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Compostos Orgânicos / Demência Frontotemporal / Inibidores de Histona Desacetilases / Haploinsuficiência / Progranulinas Tipo de estudo: Clinical_trials / Prognostic_studies / Risk_factors_studies Limite: Adult / Aged / Female / Humans / Male / Middle aged Idioma: En Revista: JAMA Netw Open Ano de publicação: 2021 Tipo de documento: Article
Texto completo
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Compostos Orgânicos / Demência Frontotemporal / Inibidores de Histona Desacetilases / Haploinsuficiência / Progranulinas Tipo de estudo: Clinical_trials / Prognostic_studies / Risk_factors_studies Limite: Adult / Aged / Female / Humans / Male / Middle aged Idioma: En Revista: JAMA Netw Open Ano de publicação: 2021 Tipo de documento: Article