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ArticlesPeer ReviewedFull Text
Hereditary transthyretin amyloid neuropathies: advances in pathophysiology, biomarkers, and treatment
Author / Creator:Adams, David ; Sekijima, Yoshiki ; Conceição, Isabel ; Waddington-Cruz, Marcia ; Polydefkis, Michael ; Echaniz-Laguna, Andoni ; Reilly, Mary M
Publisher:England: Elsevier Ltd
Journal title:Lancet neurology, 2023-11, Vol.22 (11), p.1061-1074
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Symptoms of Hereditary Transthyretin Amyloidosis: The Patient and Physician Perspective
Author / Creator:Lane, Michael ; Polydefkis, Michael
Publisher:Cheshire: Springer Healthcare
Journal title:Neurology and therapy, 2024-12, Vol.13 (6), p.1527-1533
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ArticlesPeer ReviewedFull Text
Expert opinion on monitoring symptomatic hereditary transthyretin-mediated amyloidosis and assessment of disease progression
Author / Creator:Adams, David ; Algalarrondo, Vincent ; Polydefkis, Michael ; Sarswat, Nitasha ; Slama, Michel S. ; Nativi-Nicolau, Jose
Publisher:England: BioMed Central Ltd
Journal title:Orphanet journal of rare diseases, 2021-10, Vol.16 (1), p.411-411, Article 411
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Long-term efficacy and safety of inotersen for hereditary transthyretin amyloidosis: NEURO-TTR open-label extension 3-year update
Author / Creator:Brannagan, Thomas H. ; Coelho, Teresa ; Wang, Annabel K. ; Polydefkis, Michael J. ; Dyck, Peter J. ; Berk, John L. ; Drachman, Brian ; Gorevic, Peter ; Whelan, Carol ; CONCEICAO, ISABEL ; Plante-Bordeneuve, Violaine ; Merlini, Giampaolo ; Obici, Laura ; Plana, Josep Maria Campistol ; Gamez, Josep ; Kristen, Arnt V. ; Mazzeo, Anna ; Gentile, Luca ; Narayana, Arvind ; Olugemo, Kemi ; Aquino, Peter ; Benson, Merrill D. ; Gertz, Morie
Publisher:Berlin/Heidelberg: Springer Nature
Journal title:Journal of neurology, 2022-12, Vol.269 (12), p.6416-6427
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Ethoxyquin is neuroprotective and partially prevents somatic and autonomic neuropathy in db/db mouse model of type 2 diabetes
Author / Creator:Liu, Ying ; Sun, Yuan ; Ewaleifoh, Osefame ; Wei, Josh ; Mi, Ruifa ; Zhu, Jing ; Hoke, Ahmet ; Polydefkis, Michael
Publisher:London: Nature Publishing Group UK
Journal title:Scientific reports, 2021-05, Vol.11 (1), p.10749-10749, Article 10749
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A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis
Author / Creator:Coelho, Teresa ; Adams, David ; Conceição, Isabel ; Waddington-Cruz, Márcia ; Schmidt, Hartmut H. ; Buades, Juan ; Campistol, Josep ; Berk, John L. ; Polydefkis, Michael ; Wang, Jing Jing ; Chen, Jihong ; Sweetser, Marianne T. ; Gollob, Jared ; Suhr, Ole B.
Publisher:London: BioMed Central Ltd
Journal title:Orphanet journal of rare diseases, 2020-07, Vol.15 (1), p.1-179, Article 179
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Newsletter articlesFull Text
WIPO PUBLISHES PATENT OF THE JOHNS HOPKINS UNIVERSITY, SLUSHER BARBARA S., POLYDEFKIS MICHAEL, ROJAS CAMILO, STATHIS MARIGO FOR "QUANTITATION OF GCP ACTIVITY IN BIOLOGICAL SAMPLES" (AMERICAN INVENTORS...
Publisher:Washington, D.C: HT Digital Streams Limited
Journal title:US Fed News Service, Including US State News, 2012
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Effect of diabetes type on long‐term outcome of epidermal axon regeneration
Author / Creator:Khoshnoodi, Mohammad ; Truelove, Shaun ; Polydefkis, Michael
Publisher:United States: John Wiley & Sons, Inc
Journal title:Annals of clinical and translational neurology, 2019-10, Vol.6 (10), p.2088-2096
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ArticlesPeer ReviewedFull Text
Inotersen preserves or improves quality of life in hereditary transthyretin amyloidosis
Author / Creator:Coelho, Teresa ; Yarlas, Aaron ; Waddington-Cruz, Marcia ; White, Michelle K. ; Sikora Kessler, Asia ; Lovley, Andrew ; Pollock, Michael ; Guthrie, Spencer ; Ackermann, Elizabeth J. ; Hughes, Steven G. ; Karam, Chafic ; Khella, Sami ; Gertz, Morie ; Merlini, Giampaolo ; Obici, Laura ; Schmidt, Hartmut H. ; Polydefkis, Michael ; Dyck, P. James B. ; Brannagan III, Thomas H. ; Conceição, Isabel ; Benson, Merrill D. ; Berk, John L.
Publisher:Berlin/Heidelberg: Springer Berlin Heidelberg
Journal title:Journal of neurology, 2020-04, Vol.267 (4), p.1070-1079
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ArticlesPeer ReviewedFull Text
Loss of intraepidermal nerve fiber density during SIV peripheral neuropathy is mediated by monocyte activation and elevated monocyte chemotactic proteins
Author / Creator:Lakritz, Jessica R. ; Robinson, Jake A. ; Polydefkis, Michael J. ; Miller, Andrew D. ; Burdo, Tricia H.
Publisher:England: BioMed Central Ltd
Journal title:Journal of neuroinflammation, 2015-12, Vol.12 (1), p.237-237, Article 237
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ArticlesPeer ReviewedFull Text
A phase 2a study investigating the effects of ritlecitinib on brainstem auditory evoked potentials and intraepidermal nerve fiber histology in adults with alopecia areata
Author / Creator:Anderson, Samira ; Cavaletti, Guido ; Hood, Linda J. ; Polydefkis, Michael ; Herrmann, David N. ; Rance, Gary ; King, Brett ; McMichael, Amy J. ; Senna, Maryanne M. ; Kim, Brian S. ; Napatalung, Lynne ; Wolk, Robert ; Zwillich, Samuel H. ; Schaefer, Gregor ; Gong, Yankun ; Sisson, Melanie ; Posner, Holly B.
Publisher:United States: John Wiley & Sons, Inc
Journal title:Pharmacology research & perspectives, 2024-08, Vol.12 (4), p.e1204-n/a
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ArticlesPeer ReviewedFull Text
Cutaneous Collateral Axonal Sprouting Re-Innervates the Skin Component and Restores Sensation of Denervated Swine Osteomyocutaneous Alloflaps
Author / Creator:Ibrahim, Zuhaib ; Ebenezer, Gigi ; Christensen, Joani M. ; Sarhane, Karim A. ; Hauer, Peter ; Cooney, Damon S. ; Sacks, Justin M. ; Schneeberger, Stefan ; Lee, W. P. Andrew ; Polydefkis, Michael ; Brandacher, Gerald
Publisher:United States: Public Library of Science
Journal title:PloS one, 2013-10, Vol.8 (10), p.e77646-e77646
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ArticlesPeer ReviewedFull Text
Transdermal Fentanyl Reduces Pain and Improves Functional Activity in Neuropathic Pain States
Author / Creator:Agarwal, Shefali ; Polydefkis, Michael ; Block, Brian ; Haythornthwaite, Jennifer ; Raja, Srinivasa N.
Publisher:Malden, USA: Blackwell Publishing Inc
Journal title:Pain medicine (Malden, Mass.), 2007-10, Vol.8 (7), p.554-562
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ArticlesPeer ReviewedFull Text
Glutamate carboxypeptidase activity in human skin biopsies as a pharmacodynamic marker for clinical studies
Author / Creator:Rojas, Camilo ; Stathis, Marigo ; Polydefkis, Michael ; Rudek, Michelle A ; Zhao, Ming ; Ebenezer, Gigi J ; Slusher, Barbara S
Publisher:England: BioMed Central Ltd
Journal title:Journal of translational medicine, 2011-03, Vol.9 (1), p.27-27, Article 27
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ArticlesPeer ReviewedFull Text
Correction to: Peripheral Neuropathy: No Longer the Land of Therapeutic Nihilism
Author / Creator:Polydefkis, Michael ; Sumner, Charlotte J.
Publisher:Cham: Springer International Publishing
Journal title:Neurotherapeutics, 2022-03, Vol.19 (2), p.682-682
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ArticlesPeer ReviewedFull Text
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
Author / Creator:Adams, David ; Gonzalez-Duarte, Alejandra ; O’Riordan, William D ; Yang, Chih-Chao ; Ueda, Mitsuharu ; Kristen, Arnt V ; Tournev, Ivailo ; Schmidt, Hartmut H ; Coelho, Teresa ; Berk, John L ; Lin, Kon-Ping ; Vita, Giuseppe ; Attarian, Shahram ; Planté-Bordeneuve, Violaine ; Mezei, Michelle M ; Campistol, Josep M ; Buades, Juan ; Brannagan, Thomas H ; Kim, Byoung J ; Oh, Jeeyoung ; Parman, Yesim ; Sekijima, Yoshiki ; Hawkins, Philip N ; Solomon, Scott D ; Polydefkis, Michael ; Dyck, Peter J ; Gandhi, Pritesh J ; Goyal, Sunita ; Chen, Jihong ; Strahs, Andrew L ; Nochur, Saraswathy V ; Sweetser, Marianne T ; Garg, Pushkal P ; Vaishnaw, Akshay K ; Gollob, Jared A ; Suhr, Ole B
Publisher:United States: Massachusetts Medical Society
Journal title:The New England journal of medicine, 2018-07, Vol.379 (1), p.11-21
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ArticlesPeer ReviewedFull Text
Peripheral Neuropathy: No Longer the Land of Therapeutic Nihilism
Author / Creator:Polydefkis, Michael ; Sumner, Charlotte J.
Publisher:Cham: Springer International Publishing
Journal title:Neurotherapeutics, 2021-10, Vol.18 (4), p.2153-2155
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ArticlesPeer ReviewedFull Text
Oligonucleotide Drugs for Transthyretin Amyloidosis
Author / Creator:Adams, David ; Hawkins, Philip N ; Polydefkis, Michael
Publisher:United States
Journal title:The New England journal of medicine, 2018-11, Vol.379 (21), p.2086
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Clinical and genetic profile of patients enrolled in the Transthyretin Amyloidosis Outcomes Survey (THAOS): 14-year update
Author / Creator:Dispenzieri, Angela ; Coelho, Teresa ; Conceição, Isabel ; Waddington-Cruz, Márcia ; Wixner, Jonas ; Kristen, Arnt V. ; Rapezzi, Claudio ; Planté-Bordeneuve, Violaine ; Gonzalez-Moreno, Juan ; Maurer, Mathew S. ; Grogan, Martha ; Chapman, Doug ; Amass, Leslie ; Pavia, Pablo Garcia ; Tarnev, Ivaylo ; Costello, Jose Gonzalez ; Briseno, Maria Alejandra Gonzalez Duarte ; Schmidt, Hartmut ; Drachman, Brian ; Barroso, Fabio Adrian ; Yamashita, Taro ; Lairez, Olivier ; Sekijima, Yoshiki ; Vita, Giuseppe ; Jeon, Eun-Seok ; Hanna, Mazen ; Slosky, David ; Luigetti, Marco ; LoRusso, Samantha ; Beamud, Francisco Munoz ; Adams, David ; Moelgaard, Henning ; Press, Rayomand ; Cirami, Calogero Lino ; Nienhuis, Hans ; Plana, Josep Maria Campistol ; Inamo, Jocelyn ; Jacoby, Daniel ; Emdin, Michele ; Quan, Dianna ; Hummel, Scott ; Witteles, Ronald ; Dori, Amir ; Shah, Sanjiv ; Lenihan, Daniel ; Azevedo, Olga ; Murali, Srinivas ; Zivkovic, Sasa ; Low, Soon Chai ; Nativi-Nicolau, Jose ; Fine, Nowell ; Tallaj, Jose ; Tschoepe, Carsten ; Torrón, Roberto Fernandéz ; Polydefkis, Michael ; Merlini, Giampaolo ; Badelita, Sorina ; Gottlieb, Stephen ; Tauras, James ; Correia, Edileide Barros ; Ventura, Hector ; Gess, Burkhard ; Darstein, Felix ; Oh, Jeeyoung ; Marburger, Tessa ; Van Cleemput, Johan ; Salutto, Valeria Lujan ; Parman, Yesim ; Chao, Chi-Chao ; Sarswat, Nitasha ; Mueller, Christopher ; Steidley, David ; Ralph, Jeffrey ; Warner, Alberta ; Cotts, William ; Hoffman, James ; Rugiero, Marcelo ; Misawa, Sonoko ; Blanco, Jose Luis Munoz ; Davila, Lucia Galan ; Sadeh, Menachem ; Luo, Jin ; Kyriakides, Theodoros ; Wang, Annabel ; Kaufmann, Horacio
Publisher:London: BioMed Central Ltd
Journal title:Orphanet journal of rare diseases, 2022-06, Vol.17 (1), p.1-236, Article 236
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ArticlesPeer ReviewedFull Text
Analysis of autonomic outcomes in APOLLO, a phase III trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis
Author / Creator:González-Duarte, Alejandra ; Berk, John L. ; Quan, Dianna ; Mauermann, Michelle L. ; Schmidt, Hartmut H. ; Polydefkis, Michael ; Waddington-Cruz, Márcia ; Ueda, Mitsuharu ; Conceição, Isabel M. ; Kristen, Arnt V. ; Coelho, Teresa ; Cauquil, Cécile A. ; Tard, Céline ; Merkel, Madeline ; Aldinc, Emre ; Chen, Jihong ; Sweetser, Marianne T. ; Wang, Jing Jing ; Adams, David
Publisher:Berlin/Heidelberg: Springer Berlin Heidelberg
Journal title:Journal of neurology, 2020-03, Vol.267 (3), p.703-712
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Hereditary transthyretin amyloid neuropathies: advances in pathophysiology, biomarkers, and treatment

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Symptoms of Hereditary Transthyretin Amyloidosis: The Patient and Physician Perspective

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Expert opinion on monitoring symptomatic hereditary transthyretin-mediated amyloidosis and assessment of disease progression

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Long-term efficacy and safety of inotersen for hereditary transthyretin amyloidosis: NEURO-TTR open-label extension 3-year update

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Ethoxyquin is neuroprotective and partially prevents somatic and autonomic neuropathy in db/db mouse model of type 2 diabetes

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A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis

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WIPO PUBLISHES PATENT OF THE JOHNS HOPKINS UNIVERSITY, SLUSHER BARBARA S., POLYDEFKIS MICHAEL, ROJAS CAMILO, STATHIS MARIGO FOR "QUANTITATION OF GCP ACTIVITY IN BIOLOGICAL SAMPLES" (AMERICAN INVENTORS...

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Effect of diabetes type on long‐term outcome of epidermal axon regeneration

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Inotersen preserves or improves quality of life in hereditary transthyretin amyloidosis

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Loss of intraepidermal nerve fiber density during SIV peripheral neuropathy is mediated by monocyte activation and elevated monocyte chemotactic proteins

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A phase 2a study investigating the effects of ritlecitinib on brainstem auditory evoked potentials and intraepidermal nerve fiber histology in adults with alopecia areata

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Cutaneous Collateral Axonal Sprouting Re-Innervates the Skin Component and Restores Sensation of Denervated Swine Osteomyocutaneous Alloflaps

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Transdermal Fentanyl Reduces Pain and Improves Functional Activity in Neuropathic Pain States

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Glutamate carboxypeptidase activity in human skin biopsies as a pharmacodynamic marker for clinical studies

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Correction to: Peripheral Neuropathy: No Longer the Land of Therapeutic Nihilism

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Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

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Peripheral Neuropathy: No Longer the Land of Therapeutic Nihilism

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Oligonucleotide Drugs for Transthyretin Amyloidosis

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Clinical and genetic profile of patients enrolled in the Transthyretin Amyloidosis Outcomes Survey (THAOS): 14-year update

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Analysis of autonomic outcomes in APOLLO, a phase III trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis

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