Research output per year
Research output per year
- 1 - 50 out of 121 results
Search results
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2024
Coexistence of Light Chain and Transthyretin Cardiac Amyloidosis
Gami, A., Woller, J., Scheel, P., Ali, S. A., Huff, C. A., Steenbergen, C., Halushka, M., Sharma, K., Polydefkis, M. & Vaishnav, J., Apr 3 2024, In: JACC: Case Reports. 29, 7, 102285.Research output: Contribution to journal › Article › peer-review
Open Access -
Treatment response and neurofilament light chain levels with long-term patisiran in hereditary transthyretin-mediated amyloidosis with polyneuropathy: 24-month results of an open-label extension study
Ticau, S., Aldinc, E., Polydefkis, M., Adams, D., Coelho, T., Ueda, M., Hale, C., Vest, J. & Nioi, P., 2024, In: Amyloid. 31, 1, p. 1-11 11 p.Research output: Contribution to journal › Article › peer-review
Open Access -
2023
Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial
The HELIOS-A Collaborators, 2023, In: Amyloid. 30, 1, p. 18-26 9 p.Research output: Contribution to journal › Article › peer-review
Open Access -
Hereditary transthyretin amyloid neuropathies: advances in pathophysiology, biomarkers, and treatment
Adams, D., Sekijima, Y., Conceição, I., Waddington-Cruz, M., Polydefkis, M., Echaniz-Laguna, A. & Reilly, M. M., Nov 2023, In: The Lancet Neurology. 22, 11, p. 1061-1074 14 p.Research output: Contribution to journal › Review article › peer-review
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Impact of Vutrisiran on Quality of Life and Physical Function in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy
the HELIOS-A Collaborators Study Group, Oct 2023, In: Neurology and Therapy. 12, 5, p. 1759-1775 17 p.Research output: Contribution to journal › Article › peer-review
Open Access -
Phenotypes Associated With the Val122Ile, Leu58His, and Late-Onset Val30Met Variants in Patients With Hereditary Transthyretin Amyloidosis
Zampino, S., Sheikh, F. H., Vaishnav, J., Judge, D., Pan, B., Daniel, A., Brown, E., Ebenezer, G. & Polydefkis, M., May 9 2023, In: Neurology. 100, 19, p. E2036-E2044Research output: Contribution to journal › Article › peer-review
Open Access -
2022
Clinical and genetic profile of patients enrolled in the Transthyretin Amyloidosis Outcomes Survey (THAOS): 14-year update
The THAOS investigators, Dec 2022, In: Orphanet journal of rare diseases. 17, 1, 236.Research output: Contribution to journal › Article › peer-review
Open Access -
Correction to: Peripheral Neuropathy: No Longer the Land of Therapeutic Nihilism (Neurotherapeutics, (2021), 18, 4, (2153-2155), 10.1007/s13311-021-01177-5)
Polydefkis, M. & Sumner, C. J., Mar 2022, In: Neurotherapeutics. 19, 2, p. 682 1 p.Research output: Contribution to journal › Comment/debate › peer-review
Open Access -
Large Pleural Effusion: A Pitfall in the Quantitation of 99mTc-PYP Imaging for ATTR Cardiac Amyloidosis
Khatri, W., Polydefkis, M., Vaishnav, J., Saad, E., Solnes, L. B. & Rowe, S. P., Sep 1 2022, In: Clinical nuclear medicine. 47, 9, p. E594-E595Research output: Contribution to journal › Editorial › peer-review
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Long-term efficacy and safety of inotersen for hereditary transthyretin amyloidosis: NEURO-TTR open-label extension 3-year update
for the NEURO-T. T. R. Open-Label Extension Investigators, Dec 2022, In: Journal of neurology. 269, 12, p. 6416-6427 12 p.Research output: Contribution to journal › Article › peer-review
Open Access -
Mycobacterium leprae induces Schwann cell proliferation and migration in a denervated milieu following intracutaneous excision axotomy in nine-banded armadillos
Ebenezer, G. J., Pena, M. T., Daniel, A. S., Truman, R. W., Adams, L., Duthie, M. S., Wagner, K., Zampino, S., Tolf, E., Tsottles, D. & Polydefkis, M., Jun 2022, In: Experimental Neurology. 352, 114053.Research output: Contribution to journal › Article › peer-review
Open Access -
Sex Differences in Wild-Type Transthyretin Amyloidosis: An Analysis from the Transthyretin Amyloidosis Outcomes Survey (THAOS)
The THAOS investigators, Sep 2022, In: Cardiology and Therapy. 11, 3, p. 393-405 13 p.Research output: Contribution to journal › Article › peer-review
Open Access -
2021
Ethoxyquin is neuroprotective and partially prevents somatic and autonomic neuropathy in db/db mouse model of type 2 diabetes
Liu, Y., Sun, Y., Ewaleifoh, O., Wei, J., Mi, R., Zhu, J., Hoke, A. & Polydefkis, M., Dec 2021, In: Scientific reports. 11, 1, 10749.Research output: Contribution to journal › Article › peer-review
Open Access -
Expert opinion on monitoring symptomatic hereditary transthyretin-mediated amyloidosis and assessment of disease progression
Adams, D., Algalarrondo, V., Polydefkis, M., Sarswat, N., Slama, M. S. & Nativi-Nicolau, J., Oct 3 2021, In: Orphanet journal of rare diseases. 16, 1, p. 411 1 p.Research output: Contribution to journal › Article › peer-review
Open Access -
Large Mitochondrial DNA Deletions in HIV Sensory Neuropathy
Roda, R. H., Bargiela, D., Chen, W., Perry, K., Ellis, R. J., Clifford, D. B., Bharti, A., Kallianpur, A. R., Oliveira, M. F., Diaz, M. M., Rubin, L. H., Gavegnano, C., Mcarthur, J. C., Hoke, A. & Polydefkis, M., Jul 13 2021, In: Neurology. 97, 2, p. E156-E165Research output: Contribution to journal › Article › peer-review
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Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study
patisiran Global OLE study group, Jan 2021, In: The Lancet Neurology. 20, 1, p. 49-59 11 p.Research output: Contribution to journal › Article › peer-review
5 Scopus citations -
Neurofilament Light Chain as a Biomarker of Hereditary Transthyretin-Mediated Amyloidosis
Ticau, S., Sridharan, G. V., Tsour, S., Cantley, W. L., Chan, A., Gilbert, J. A., Erbe, D., Aldinc, E., Reilly, M. M., Adams, D., Polydefkis, M., Fitzgerald, K., Vaishnaw, A. & Nioi, P., Jan 19 2021, In: Neurology. 96, 3, p. E412-E422Research output: Contribution to journal › Article › peer-review
Open Access4 Scopus citations -
Peripheral Neuropathy: No Longer the Land of Therapeutic Nihilism
Polydefkis, M. & Sumner, C. J., Oct 2021, In: Neurotherapeutics. 18, 4, p. 2153-2155 3 p.Research output: Contribution to journal › Review article › peer-review
Open Access -
Research design considerations for chronic pain prevention clinical trials: IMMPACT recommendations
Gewandter, J. S., Dworkin, R. H., Turk, D. C., Farrar, J. T., Fillingim, R. B., Gilron, I., Markman, J. D., Oaklander, A. L., Polydefkis, M. J., Raja, S. N., Robinson, J. P., Woolf, C. J., Ziegler, D., Ashburn, M. A., Burke, L. B., Cowan, P., George, S. Z., Goli, V., Graff, O. X., Iyengar, S., & 13 others, Jan 21 2021, In: Pain Reports. 6, 1, A211.Research output: Contribution to journal › Review article › peer-review
Open Access -
Sex-Related Risk of Cardiac Involvement in Hereditary Transthyretin Amyloidosis: Insights From THAOS
THAOS Investigators, Oct 2021, In: JACC: Heart Failure. 9, 10, p. 736-746 11 p.Research output: Contribution to journal › Article › peer-review
Open Access -
Temporal Trends of Wild-Type Transthyretin Amyloid Cardiomyopathy in the Transthyretin Amyloidosis Outcomes Survey
THAOS Investigators, Oct 2021, In: JACC: CardioOncology. 3, 4, p. 537-546 10 p.Research output: Contribution to journal › Article › peer-review
Open Access -
The diagnostic challenges of cardiac amyloidosis: A practical approach to the two main types
Varga, C., Dorbala, S., Lousada, I., Polydefkis, M. J., Wechalekar, A., Maurer, M. S. & Comenzo, R. L., Jan 2021, In: Blood Reviews. 45, 100720.Research output: Contribution to journal › Review article › peer-review
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2020
Analysis of autonomic outcomes in APOLLO, a phase III trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis
González-Duarte, A., Berk, J. L., Quan, D., Mauermann, M. L., Schmidt, H. H., Polydefkis, M., Waddington-Cruz, M., Ueda, M., Conceição, I. M., Kristen, A. V., Coelho, T., Cauquil, C. A., Tard, C., Merkel, M., Aldinc, E., Chen, J., Sweetser, M. T., Wang, J. J. & Adams, D., Mar 1 2020, In: Journal of neurology. 267, 3, p. 703-712 10 p.Research output: Contribution to journal › Article › peer-review
Open Access10 Scopus citations -
A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis
Coelho, T., Adams, D., Conceição, I., Waddington-Cruz, M., Schmidt, H. H., Buades, J., Campistol, J., Berk, J. L., Polydefkis, M., Wang, J. J., Chen, J., Sweetser, M. T., Gollob, J. & Suhr, O. B., Jul 8 2020, In: Orphanet journal of rare diseases. 15, 1, 179.Research output: Contribution to journal › Article › peer-review
Open Access7 Scopus citations -
Correction to: Analysis of autonomic outcomes in APOLLO, a phase III trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis (Journal of Neurology, (2020), 267, 3, (703-712), 10.1007/s00415-019-09602-8)
González-Duarte, A., Berk, J. L., Quan, D., Mauermann, M. L., Schmidt, H. H., Polydefkis, M., Waddington-Cruz, M., Ueda, M., Conceição, I. M., Kristen, A. V., Coelho, T., Cauquil, C. A., Tard, C., Merkel, M., Aldinc, E., Chen, J., Sweetser, M. T., Wang, J. J. & Adams, D., Mar 1 2020, In: Journal of neurology. 267, 3, p. 713-714 2 p.Research output: Contribution to journal › Comment/debate › peer-review
Open Access -
Early data on long-term efficacy and safety of inotersen in patients with hereditary transthyretin amyloidosis: a 2-year update from the open-label extension of the NEURO-TTR trial
Brannagan, T. H., Wang, A. K., Coelho, T., Waddington Cruz, M., Polydefkis, M. J., Dyck, P. J., Plante-Bordeneuve, V., Berk, J. L., Barroso, F., Merlini, G., Conceição, I., Hughes, S. G., Kwoh, J., Jung, S. W., Guthrie, S., Pollock, M., Benson, M. D., Gertz, M., Drachman, B., Gorevic, P., & 13 others, Aug 1 2020, In: European Journal of Neurology. 27, 8, p. 1374-1381 8 p.Research output: Contribution to journal › Article › peer-review
Open Access11 Scopus citations -
Idiopathic distal sensory polyneuropathy: ACTTION diagnostic criteria
Freeman, R., Gewandter, J. S., Faber, C. G., Gibbons, C., Haroutounian, S., Lauria, G., Levine, T., Malik, R. A., Singleton, J. R., Smith, A. G., Bell, J., Dworkin, R. H., Feldman, E., Herrmann, D. N., Hoke, A., Kolb, N., Mansikka, H., Oaklander, A. L., Peltier, A., Polydefkis, M., & 6 others, Dec 1 2020, In: Neurology. 95, 22, p. 1005-1014 10 p.Research output: Contribution to journal › Review article › peer-review
3 Scopus citations -
Inotersen preserves or improves quality of life in hereditary transthyretin amyloidosis
Coelho, T., Yarlas, A., Waddington-Cruz, M., White, M. K., Sikora Kessler, A., Lovley, A., Pollock, M., Guthrie, S., Ackermann, E. J., Hughes, S. G., Karam, C., Khella, S., Gertz, M., Merlini, G., Obici, L., Schmidt, H. H., Polydefkis, M., Dyck, P. J. B., Brannagan, T. H., Conceição, I., & 2 others, Apr 1 2020, In: Journal of neurology. 267, 4, p. 1070-1079 10 p.Research output: Contribution to journal › Article › peer-review
Open Access6 Scopus citations -
mNIS+7 and lower limb function in inotersen treatment of hereditary transthyretin-mediated amyloidosis
Dyck, P. J. B., Kincaid, J. C., Wiesman, J. F., Polydefkis, M., Litchy, W. J., Mauermann, M. L., Ackermann, E. J., Guthrie, S., Pollock, M., Jung, S. W., Baker, B. F. & Dyck, P. J., Oct 1 2020, In: Muscle and Nerve. 62, 4, p. 502-508 7 p.Research output: Contribution to journal › Article › peer-review
Open Access -
Monocarboxylate transporter 1 in Schwann cells contributes to maintenance of sensory nerve myelination during aging
Jha, M. K., Lee, Y., Russell, K. A., Yang, F., Dastgheyb, R. M., Deme, P., Ament, X. H., Chen, W., Liu, Y., Guan, Y., Polydefkis, M. J., Hoke, A., Haughey, N. J., Rothstein, J. D. & Morrison, B. M., Jan 1 2020, In: Glia. 68, 1, p. 161-177 17 p.Research output: Contribution to journal › Article › peer-review
Open Access15 Scopus citations -
Neuropathy symptom and change: Inotersen treatment of hereditary transthyretin amyloidosis
Dyck, P. J. B., Coelho, T., Waddington Cruz, M., Brannagan, T. H., Khella, S., Karam, C., Berk, J. L., Polydefkis, M. J., Kincaid, J. C., Wiesman, J. F., Litchy, W. J., Mauermann, M. L., Ackermann, E. J., Baker, B. F., Jung, S. W., Guthrie, S., Pollock, M. & Dyck, P. J., Oct 1 2020, In: Muscle and Nerve. 62, 4, p. 509-515 7 p.Research output: Contribution to journal › Article › peer-review
Open Access -
Pain mechanisms in hereditary palmoplantar keratodermas
Weinberg, R. L., Coulombe, P. A., Polydefkis, M. & Caterina, M. J., Mar 1 2020, In: British Journal of Dermatology. 182, 3, p. 543-551 9 p.Research output: Contribution to journal › Review article › peer-review
2 Scopus citations -
Reducing monocarboxylate transporter MCT1 worsens experimental diabetic peripheral neuropathy
Jha, M. K., Ament, X. H., Yang, F., Liu, Y., Polydefkis, M. J., Pellerin, L. & Morrison, B. M., Nov 2020, In: Experimental Neurology. 333, 113415.Research output: Contribution to journal › Article › peer-review
Open Access1 Scopus citations -
TTR gene silencing therapy in post liver transplant hereditary ATTR amyloidosis patients
Moshe-Lilie, O., Dimitrova, D., Heitner, S. B., Brannagan, T. H., Zivkovic, S., Hanna, M., Masri, A., Polydefkis, M., Berk, J. L., Gertz, M. A. & Karam, C., Dec 2020, In: Amyloid. 27, 4, p. 250-253 4 p.Research output: Contribution to journal › Article › peer-review
3 Scopus citations -
2019
An indirect treatment comparison of the efficacy of patisiran and tafamidis for the treatment of hereditary transthyretin-mediated amyloidosis with polyneuropathy
Planté-Bordeneuve, V., Lin, H., Gollob, J., Agarwal, S., Betts, M., Fahrbach, K., Chitnis, M. & Polydefkis, M., Mar 4 2019, In: Expert opinion on pharmacotherapy. 20, 4, p. 473-481 9 p.Research output: Contribution to journal › Article › peer-review
Open Access12 Scopus citations -
Development of measures of polyneuropathy impairment in hATTR amyloidosis: From NIS to mNIS + 7
Dyck, P. J. B., González-Duarte, A., Obici, L., Polydefkis, M., Wiesman, J. F., Antonino, I., Litchy, W. J. & Dyck, P. J., Oct 15 2019, In: Journal of the Neurological Sciences. 405, 116424.Research output: Contribution to journal › Review article › peer-review
Open Access15 Scopus citations -
Effect of diabetes type on long-term outcome of epidermal axon regeneration
Khoshnoodi, M., Truelove, S. & Polydefkis, M., Oct 1 2019, In: Annals of Clinical and Translational Neurology. 6, 10, p. 2088-2096 9 p.Research output: Contribution to journal › Article › peer-review
Open Access3 Scopus citations -
Response to the letter to the editor on “an indirect treatment comparison of the efficacy of patisiran and tafamidis for the treatment of hereditary transthyretin-mediated amyloidosis with polyneuropathy”
Lin, H., Agarwal, S., Betts, M., Fahrbach, K., Chitnis, M. & Polydefkis, M., 2019, In: Expert opinion on pharmacotherapy. 20, 12, p. 1529-1530 2 p.Research output: Contribution to journal › Letter › peer-review
Open Access -
2018
Correction: LepVax, a defined subunit vaccine that provides effective pre-exposure and post-exposure prophylaxis of M. leprae infection (npj Vaccines (2018) DOI: 10.1038/s41541-018-0050-z)
Duthie, M. S., Pena, M. T., Ebenezer, G. J., Gillis, T. P., Sharma, R., Cunningham, K., Polydefkis, M., Maeda, Y., Makino, M., Truman, R. W. & Reed, S. G., Dec 1 2018, In: npj Vaccines. 3, 1, 55.Research output: Contribution to journal › Comment/debate › peer-review
Open Access -
Ghrelin agonist HM01 attenuates chemotherapy-induced neurotoxicity in rodent models
Chiorazzi, A., Wozniak, K. M., Rais, R., Wu, Y., Gadiano, A. J., Farah, M. H., Liu, Y., Canta, A., Alberti, P., Rodriguez-Menendez, V., Meregalli, C., Fumagalli, G., Monza, L., Pozzi, E., Vornov, J. J., Polydefkis, M., Pietra, C., Slusher, B. S. & Cavaletti, G., Dec 5 2018, In: European Journal of Pharmacology. 840, p. 89-103 15 p.Research output: Contribution to journal › Article › peer-review
4 Scopus citations -
Hereditary transthyretin amyloidosis: baseline characteristics of patients in the NEURO-TTR trial
Waddington-Cruz, M., Ackermann, E. J., Polydefkis, M., Heitner, S. B., Dyck, P. J., Barroso, F. A., Wang, A. K., Berk, J. L., Dyck, P. J. B., Monia, B. P., Hughes, S. G., Tai, L., Jesse Kwoh, T., Jung, S. W., Coelho, T., Benson, M. D. & Gertz, M. A., Jul 3 2018, In: Amyloid. 25, 3, p. 180-188 9 p.Research output: Contribution to journal › Article › peer-review
Open Access8 Scopus citations -
Inotersen treatment for patients with Hereditary transthyretin amyloidosis
Benson, M. D., Waddington-Cruz, M., Berk, J. L., Polydefkis, M., Dyck, P. J., Wang, A. K., Planté-Bordeneuve, V., Barroso, F. A., Merlini, G., Obici, L., Scheinberg, M., Brannagan, T. H., Litchy, W. J., Whelan, C., Drachman, B. M., Adams, D., Heitner, S. B., Conceição, I., Schmidt, H. H., Vita, G., & 15 others, Jul 5 2018, In: New England Journal of Medicine. 379, 1, p. 22-31 10 p.Research output: Contribution to journal › Article › peer-review
Open Access462 Scopus citations -
LepVax, a defined subunit vaccine that provides effective pre-exposure and post-exposure prophylaxis of M. leprae infection
Duthie, M. S., Pena, M. T., Ebenezer, G. J., Gillis, T. P., Sharma, R., Cunningham, K., Polydefkis, M., Maeda, Y., Makino, M., Truman, R. W. & Reed, S. G., Dec 1 2018, In: npj Vaccines. 3, 1, 12.Research output: Contribution to journal › Article › peer-review
Open Access22 Scopus citations -
Nociceptin/orphanin FQ opioid peptide-receptor expression in pachyonychia congenita
Pan, B., Schröder, W., Jostock, R., Schwartz, M., Rosson, G. & Polydefkis, M., Dec 2018, In: Journal of the Peripheral Nervous System. 23, 4, p. 241-248 8 p.Research output: Contribution to journal › Article › peer-review
6 Scopus citations -
Oligonucleotide Drugs for Transthyretin Amyloidosis
Adams, D., Hawkins, P. N. & Polydefkis, M., Nov 22 2018, In: The New England journal of medicine. 379, 21, p. 2086 1 p.Research output: Contribution to journal › Letter › peer-review
5 Scopus citations -
Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis
Adams, D., Gonzalez-Duarte, A., O'Riordan, W. D., Yang, C. C., Ueda, M., Kristen, A. V., Tournev, I., Schmidt, H. H., Coelho, T., Berk, J. L., Lin, K. P., Vita, G., Attarian, S., Planté-Bordeneuve, V., Mezei, M. M., Campistol, J. M., Buades, J., Brannagan, T. H., Kim, B. J., Oh, J., & 16 others, Jul 5 2018, In: New England Journal of Medicine. 379, 1, p. 11-21 11 p.Research output: Contribution to journal › Article › peer-review
Open Access797 Scopus citations -
Peripheral neuropathy induced by microtubule-targeted chemotherapies: Insights into acute injury and long-term recovery
Wozniak, K. M., Vornov, J. J., Wu, Y., Liu, Y., Carozzi, V. A., Rodriguez-Menendez, V., Ballarini, E., Alberti, P., Pozzi, E., Semperboni, S., Cook, B. M., Littlefield, B. A., Nomoto, K., Condon, K., Eckley, S., DesJardins, C., Wilson, L., Jordan, M. A., Feinstein, S. C., Cavaletti, G., & 2 others, Feb 1 2018, In: Cancer Research. 78, 3, p. 817-829 13 p.Research output: Contribution to journal › Article › peer-review
Open Access30 Scopus citations -
Reply: Non-freezing cold injury: A multi-faceted syndrome
Vale, T. A., Symmonds, M., Polydefkis, M., Byrnes, K., Rice, A. S. C., Themistocleous, A. C. & Bennett, D. L. H., Feb 1 2018, In: Brain. 141, 2, p. e10Research output: Contribution to journal › Letter › peer-review
Open Access -
2017
An oral form of methylglyoxal-bis-guanylhydrazone reduces monocyte activation and traffic to the dorsal root ganglia in a primate model of HIV-peripheral neuropathy
Lakritz, J. R., Yalamanchili, S., Polydefkis, M. J., Miller, A. D., McGrath, M. S., Williams, K. C. & Burdo, T. H., Aug 1 2017, In: Journal of neurovirology. 23, 4, p. 568-576 9 p.Research output: Contribution to journal › Article › peer-review
4 Scopus citations -
Assessing mNIS+7Ionis and international neurologists' proficiency in a familial amyloidotic polyneuropathy trial
Dyck, P. J., Kincaid, J. C., Dyck, P. J. B., Chaudhry, V., Goyal, N. A., Alves, C., Salhi, H., Wiesman, J. F., Labeyrie, C., Robinson-Papp, J., Cardoso, M., Laura, M., Ruzhansky, K., Cortese, A., Brannagan, T. H., Khoury, J., Khella, S., Waddington-Cruz, M., Ferreira, J., Wang, A. K., & 24 others, Nov 2017, In: Muscle and Nerve. 56, 5, p. 901-911 11 p.Research output: Contribution to journal › Article › peer-review
29 Scopus citations