Zobrazeno 1 - 10
of 32
pro vyhledávání: '"Elizabeth J, Ackermann"'
Autor:
Christoph J Niemietz, Vanessa Sauer, Jacqueline Stella, Lutz Fleischhauer, Gursimran Chandhok, Sarah Guttmann, Yesim Avsar, Shuling Guo, Elizabeth J Ackermann, Jared Gollob, Brett P Monia, Andree Zibert, Hartmut H-J Schmidt
Publikováno v:
PLoS ONE, Vol 11, Iss 9, p e0161455 (2016)
Familial amyloid polyneuropathy (FAP) is caused by mutations of the transthyretin (TTR) gene, predominantly expressed in the liver. Two compounds that knockdown TTR, comprising a small interfering RNA (siRNA; ALN-TTR-02) and an antisense oligonucleot
Externí odkaz:
https://doaj.org/article/1796834c54db428483d9067e5e8d8e11
Autor:
Janice F. Wiesman, Michael Polydefkis, William J. Litchy, Michelle L. Mauermann, P. James B. Dyck, Shiangtung W. Jung, Brenda F. Baker, Michael L. Pollock, Spencer D. Guthrie, John C. Kincaid, Peter J. Dyck, Elizabeth J. Ackermann
Publikováno v:
Muscle & Nerve
Introduction Inotersen, an antisense oligonucleotide inhibitor of transthyretin (TTR) protein production, demonstrated significant benefit versus placebo in the modified Neuropathy Impairment Score (NIS) +7 neurophysiologic tests (mNIS+7) in patients
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4ce6ccb7b7d8b3821ff0e8b9fb3f3eaf
https://doi.org/10.1002/mus.27022
https://doi.org/10.1002/mus.27022
Autor:
William J. Litchy, Michael L. Pollock, P. James B. Dyck, Michelle L. Mauermann, Teresa Coelho, Chafic Karam, Michael Polydefkis, Sami Khella, Janice F. Wiesman, Thomas H. Brannagan, Márcia Waddington Cruz, Spencer D. Guthrie, John L. Berk, Shiangtung W. Jung, Peter J. Dyck, Brenda F. Baker, Elizabeth J. Ackermann, John C. Kincaid
Publikováno v:
Muscle & Nerve
Introduction Hereditary transthyretin‐mediated amyloidosis (hATTR) manifests as multisystem dysfunction, including progressive polyneuropathy. Inotersen, an antisense oligonucleotide, improved the course of neuropathic impairment in patients with h
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f21cc65dcadeab19400bdedb50e5f161
https://doi.org/10.1002/mus.27023
https://doi.org/10.1002/mus.27023
Autor:
Jon W Collins, Scott P. Henry, Richard S. Geary, Brett P. Monia, Elizabeth J. Ackermann, Shannon Hall, Yanfeng Wang, Rosie Z. Yu
Publikováno v:
Nucleic Acid Therapeutics
A population pharmacokinetic (PK) and pharmacodynamic (PD) model was developed for inotersen to evaluate exposure–response relationships and to optimize therapeutic dosing regimen in patients with hereditary transthyretin (TTR) amyloidosis polyneur
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::7738a399c01da5a8eb9f6c5e8d9fd228
https://doi.org/10.1089/nat.2019.0822
https://doi.org/10.1089/nat.2019.0822
Autor:
Morie A. Gertz, Asia Sikora Kessler, Laura Obici, Andrew Lovley, Michael L. Pollock, Aaron Yarlas, Michelle K. White, Pankaj A. Patel, Spencer D. Guthrie, Elizabeth J. Ackermann, Noel R. Dasgupta
Publikováno v:
Muscle & Nerve
Introduction Hereditary transthyretin (hATTR) amyloidosis is a progressive, degenerative disease, with peripheral neuropathy, cardiomyopathy, and other clinical manifestations. In this study we examine the impact of hATTR amyloidosis on quality of li
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c2976649555e09ef2deb8ddb164c3c87
https://doi.org/10.1002/mus.26515
https://doi.org/10.1002/mus.26515
Autor:
Asia Sikora Kessler, Thomas H. Brannagan, Laura Obici, Sami Khella, Andrew Lovley, Hartmut Schmidt, Steven G. Hughes, Michelle K. White, Michael Polydefkis, Teresa Coelho, Morie A. Gertz, Giampaolo Merlini, Michael L. Pollock, Spencer D. Guthrie, John L. Berk, P. James B. Dyck, Aaron Yarlas, Chafic Karam, Márcia Waddington-Cruz, Elizabeth J. Ackermann, Isabel Conceição, Merrill D. Benson
Publikováno v:
Journal of Neurology
Objective To examine the impact on quality of life (QOL) of patients with hATTR amyloidosis with polyneuropathy treated with inotersen (Tegsedi™) versus placebo. Methods Data were from the NEURO-TTR trial (ClinicalTrials.gov Identifier: NCT01737398
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ea301c4808ed94febe373f953d68a57b
https://hdl.handle.net/10400.16/2665
https://hdl.handle.net/10400.16/2665
Autor:
Marcus V. Pinto, Márcia Waddington-Cruz, Linde E. Gove, Steven G. Hughes, Bryan M. McCauley, P. James B. Dyck, Elizabeth J. Ackermann, Peter J. Dyck
Publikováno v:
Journal of the Neurological Sciences. 394:78-83
Objective Report on the kind and distribution of somatotopic sensation loss and its utility in assessing severity of sensation loss in study of a large international cohort of patients with hereditary transthyretin amyloidosis with polyneuropathy (hA
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::bae1f1b47e6a2ad8890b9de63fb37c5e
https://doi.org/10.1016/j.jns.2018.08.031
https://doi.org/10.1016/j.jns.2018.08.031
Autor:
T. Jesse Kwoh, Merrill D. Benson, Teresa Coelho, Brett P. Monia, Steven G. Hughes, John L. Berk, Annabel K. Wang, Stephen B. Heitner, Peter J. Dyck, Michael Polydefkis, Elizabeth J. Ackermann, Shiangtung W. Jung, Fabio Barroso, Li Tai, Morie A. Gertz, P. James B. Dyck, Márcia Waddington-Cruz
Publikováno v:
Amyloid. 25:180-188
Hereditary transthyretin (ATTRm) amyloidosis is a rare, progressive and fatal disease with a range of clinical manifestations.This study comprehensively evaluates disease characteristics in a large, diverse cohort of patients with ATTRm amyloidosis.A
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::dd9c81cd08803b7cb196351d4559093d
https://doi.org/10.1080/13506129.2018.1503593
https://doi.org/10.1080/13506129.2018.1503593
Autor:
Karen A. Lodermeier, Thomas H. Brannagan, Joao Vasco Ferreira, Steven G. Hughes, Violaine Planté-Bordeneuve, Rickey E. Carter, Jessica Robinson-Papp, Rito Bergemann, Peter J. Dyck, Christina Alves, Julie Khoury, John C. Kincaid, John L. Berk, Matilde Laura, Samar S. Ayache, Carol J. Whelan, Brian M. Drachman, Isabel Conceição, Merrill D. Benson, Sami Khella, Michael Polydefkis, Teresa Coelho, Morie A. Gertz, William J. Litchy, Hayet Salhi, Vinay Chaudhry, Stephen B. Heitner, David H. Adams, Giampaolo Merlini, Andrea Cortese, Janice F. Wiesman, P. James B. Dyck, Namita Goyal, Márcio Cardoso, Christopher J. Klein, Katherine Ruzhansky, Annabel K. Wang, Michelle L. Mauermann, Marcus V. Pinto, Márcia Waddington-Cruz, Jenny L. Davies, Céline Labeyrie, Peter D. Gorevic, Elizabeth J. Ackermann
Publikováno v:
Muscle & Nerve. 56:901-911
Introduction Polyneuropathy signs (Neuropathy Impairment Score, NIS), neurophysiologic tests (m+7Ionis ), disability, and health scores were assessed in baseline evaluations of 100 patients entered into an oligonucleotide familial amyloidotic polyneu
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::87068ae77771d9d1a58bb08875422543
https://doi.org/10.1002/mus.25563
https://doi.org/10.1002/mus.25563
Autor:
Morton A. Scheinberg, Jesse Kwoh, Edward Gane, Laura Obici, Thomas H. Brannagan, Brett P. Monia, Giampaolo Merlini, Shiangtung W. Jung, Josep Gamez, Morie A. Gertz, John L. Berk, Scott D. Solomon, Fabio Barroso, Brian M. Drachman, Márcia Waddington-Cruz, David C. Adams, Hartmut H. Schmidt, Carol J. Whelan, Violaine Planté-Bordeneuve, William J. Litchy, Peter D. Gorevic, Elizabeth J. Ackermann, Annabel K. Wang, Brenda F. Baker, Isabel Conceição, Steven G. Hughes, Merrill D. Benson, Josep M. Campistol, Bradley W. McEvoy, Michael Polydefkis, Teresa Coelho, Giuseppe Vita, Peter J. Dyck, Amil M. Shah, Stephen B. Heitner
Publikováno v:
Dipòsit Digital de la UB
Universidad de Barcelona
Recercat. Dipósit de la Recerca de Catalunya
instname
Universidad de Barcelona
Recercat. Dipósit de la Recerca de Catalunya
instname
BACKGROUND: Hereditary transthyretin amyloidosis is caused by pathogenic single-nucleotide variants in the gene encoding transthyretin ( TTR) that induce transthyretin misfolding and systemic deposition of amyloid. Progressive amyloid accumulation le
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::95fdbf6a28c8960b7f39722805188ea4
http://hdl.handle.net/2445/154868
http://hdl.handle.net/2445/154868