A Review of Tafamidis for the Treatment of Transthyretin-Related Amyloidosis

по Waddington Cruz, Márcia, Benson, Merril D.
Опубликовано в: Neurol Ther (2015)

Early intervention with tafamidis provides long-term (5.5-year) delay of neurologic progression in transthyretin hereditary amyloid polyneuropathy

по Waddington Cruz, Márcia, Amass, Leslie, Keohane, Denis, Schwartz, Jeffrey, Li, Huihua, Gundapaneni, Balarama
Опубликовано в: Amyloid (2016)

Evaluation of Mortality During Long-Term Treatment with Tafamidis for Transthyretin Amyloidosis with Polyneuropathy: Clinical Trial Results up to 8.5 Years

по Merlini, Giampaolo, Coelho, Teresa, Waddington Cruz, Márcia, Li, Huihua, Stewart, Michelle, Ebede, Ben
Опубликовано в: Neurol Ther (2020)

Early intervention with tafamidis provides long-term benefit in delaying neurological progression in patients with transthyretin familial amyloid polyneuropathy

по Waddington-Cruz, Marcia, Amass, Leslie, Keohane, Denis, Schwartz, Jeffrey, Gundapaneni, Balarama, Li, Hui Hua
Опубликовано в: Orphanet J Rare Dis (2015)

Phase 2 open-label extention (OLE) study of patisiran, an investigational siRNA agent for familial amyloidotic polyneuropathy (FAP)

по Adams, David, Suhr, Ole B, Conceicao, Isabel, Waddington-Cruz, Marcia, Schmidt, Hartmut, Buades, Juan, Campistol, Josep, Coehlo, Teresa
Опубликовано в: Orphanet J Rare Dis (2015)

Epidemiological and clinical characteristics of symptomatic hereditary transthyretin amyloid polyneuropathy: a global case series

по Waddington-Cruz, Márcia, Schmidt, Hartmut, Botteman, Marc F., Carter, John A., Stewart, Michelle, Hopps, Markay, Fallet, Shari, Amass, Leslie
Опубликовано в: Orphanet J Rare Dis (2019)

Estimating the global prevalence of transthyretin familial amyloid polyneuropathy

по Schmidt, Hartmut H., Waddington‐Cruz, Márcia, Botteman, Marc F., Carter, John A., Chopra, Avijeet S., Hopps, Markay, Stewart, Michelle, Fallet, Shari, Amass, Leslie
Опубликовано в: Muscle Nerve (2018)

Inflammatory profiling of patients with familial amyloid polyneuropathy

по Azevedo, Estefania P., Guimaraes-Costa, Anderson B., Bandeira-Melo, Christianne, Chimelli, Leila, Waddington-Cruz, Marcia, Saraiva, Elvira M., Palhano, Fernando L., Foguel, Debora
Опубликовано в: BMC Neurol (2019)

Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study

по Suhr, Ole B, Coelho, Teresa, Buades, Juan, Pouget, Jean, Conceicao, Isabel, Berk, John, Schmidt, Hartmut, Waddington-Cruz, Márcia, Campistol, Josep M., Bettencourt, Brian R., Vaishnaw, Akshay, Gollob, Jared, Adams, David
Опубликовано в: Orphanet J Rare Dis (2015)

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

по 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.
Опубликовано в: Orphanet J Rare Dis (2020)

Tafamidis for transthyretin familial amyloid polyneuropathy: A randomized, controlled trial

по Coelho, Teresa, Maia, Luis F., Martins da Silva, Ana, Waddington Cruz, Marcia, Planté-Bordeneuve, Violaine, Lozeron, Pierre, Suhr, Ole B., Campistol, Josep M., Conceição, Isabel Maria, Schmidt, Hartmut H.-J., Trigo, Pedro, Kelly, Jeffery W., Labaudinière, Richard, Chan, Jason, Packman, Jeff, Wilson, Amy, Grogan, Donna R.
Опубликовано 2012

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

по 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
Опубликовано в: J Neurol (2020)

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

по 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
Опубликовано в: J Neurol (2019)

Neuropathy symptom and change: Inotersen treatment of hereditary transthyretin amyloidosis

по Dyck, P. James B., Coelho, Teresa, Waddington Cruz, Marcia, Brannagan, Thomas H., Khella, Sami, Karam, Chafic, Berk, John L., Polydefkis, Michael J., Kincaid, John C., Wiesman, Janice F., Litchy, William J., Mauermann, Michelle L., Ackermann, Elizabeth J., Baker, Brenda F., Jung, Shiangtung W., Guthrie, Spencer, Pollock, Michael, Dyck, Peter J.
Опубликовано в: Muscle Nerve (2020)

Inotersen preserves or improves quality of life in hereditary transthyretin amyloidosis

по 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.
Опубликовано в: J Neurol (2019)

Design and Rationale of the Global Phase 3 NEURO-TTRansform Study of Antisense Oligonucleotide AKCEA-TTR-L(Rx) (ION-682884-CS3) in Hereditary Transthyretin-Mediated Amyloid Polyneu...

по Coelho, Teresa, Ando, Yukio, Benson, Merrill D., Berk, John L., Waddington-Cruz, Márcia, Dyck, Peter J., Gillmore, Julian D., Khella, Sami L., Litchy, William J., Obici, Laura, Monteiro, Cecilia, Tai, Li-Jung, Viney, Nicholas J., Buchele, Gustavo, Brambatti, Michela, Jung, Shiangtung W., St. L. O’Dea, Louis, Tsimikas, Sotirios, Schneider, Eugene, Geary, Richard S., Monia, Brett P., Gertz, Morie
Опубликовано в: Neurol Ther (2021)

ATTR amyloidosis during the COVID-19 pandemic: insights from a global medical roundtable

по Brannagan, Thomas H., Auer-Grumbach, Michaela, Berk, John L., Briani, Chiara, Bril, Vera, Coelho, Teresa, Damy, Thibaud, Dispenzieri, Angela, Drachman, Brian M., Fine, Nowell, Gaggin, Hanna K., Gertz, Morie, Gillmore, Julian D., Gonzalez, Esther, Hanna, Mazen, Hurwitz, David R., Khella, Sami L., Maurer, Mathew S., Nativi-Nicolau, Jose, Olugemo, Kemi, Quintana, Luis F., Rosen, Andrew M., Schmidt, Hartmut H., Shehata, Jacqueline, Waddington-Cruz, Marcia, Whelan, Carol, Ruberg, Frederick L.
Опубликовано в: Orphanet J Rare Dis (2021)

Assessing mNIS+7(Ionis) and International Neurologists’ Proficiency in a FAP Trial

по Dyck, Peter J., Kincaid, John C., Dyck, P. James B., Chaudhry, Vinay, Goyal, Namita A., Alves, Christina, Salhi, Hayet, Wiesman, Janice F., Labeyrie, Celine, Robinson-Papp, Jessica, Cardoso, Márcio, Laura, Matilde, Ruzhansky, Katherine, Cortese, Andrea, Brannagan, Thomas H., Khoury, Julie, Khella, Sami, Waddington-Cruz, Márcia, Ferreira, João, Wang, Annabel K., Pinto, Marcus V., Ayache, Samar S., Benson, Merrill D., Berk, John L., Coelho, Teresa, Polydefkis, Michael, Gorevic, Peter, Adams, David H., Plante-Bordeneuve, Violaine, Whelan, Carol, Merlini, Giampaolo, Heitner, Stephen, Drachman, Brian M., Conceição, Isabel, Klein, Christopher J., Gertz, Morie A., Ackermann, Elizabeth J., Hughes, Steven. G., Mauermann, Michelle. L., Bergemann, Rito, Lodermeier, Karen A., Davies, Jenny L., Carter, Rickey E., Litchy, William J.
Опубликовано в: Muscle Nerve (2017)

Minimal manifestation status and prednisone withdrawal in the MGTX trial

по Lee, Ikjae, Kuo, Hui-Chien, Aban, Inmaculada B., Cutter, Gary R., McPherson, Tarrant, Kaminski, Henry J., Sussman, Jon, Ströbel, Philipp, Oger, Joel, Cea, Gabriel, Heckmann, Jeannine M., Evoli, Amelia, Nix, Wilfred, Ciafaloni, Emma, Antonini, Giovanni, Witoonpanich, Rawiphan, King, John O., Beydoun, Said R., Chalk, Colin H., Barboi, Alexandru C., Amato, Anthony A., Shaibani, Aziz I., Katirji, Bashar, Lecky, Bryan R.F., Buckley, Camilla, Vincent, Angela, Dias-Tosta, Elza, Yoshikawa, Hiroaki, Waddington-Cruz, Marcia, Pulley, Michael T., Rivner, Michael H., Kostera-Pruszczyk, Anna, Pascuzzi, Robert M., Jackson, Carlayne E., Verschuuren, Jan J.G., Massey, Janice M., Kissel, John T., Werneck, Lineu C., Benatar, Michael, Barohn, Richard J., Tandan, Rup, Mozaffar, Tahseen, Conwit, Robin, Minisman, Greg, Sonett, Joshua R., Wolfe, Gil I.
Опубликовано в: Neurology (2020)

Long-term effect of thymectomy in patients with non-thymomatous myasthenia gravis treated with prednisone: 2-year extension of the MGTX randomised trial

по Wolfe, Gil I., Kaminski, Henry J., Aban, Inmaculada B., Minisman, Greg, Kuo, Hui-Chien, Marx, Alexander, Ströbel, Philipp, Mazia, Claudio, Oger, Joel, Gabriel Cea, J., Heckmann, Jeannine M., Evoli, Amelia, Nix, Wilfred, Ciafaloni, Emma, Antonini, Giovanni, Witoonpanich, Rawiphan, King, John O., Beydoun, Said R., Chalk, Colin H., Barboi, Alexandru C., Amato, Anthony A., Shaibani, Aziz I., Katirji, Bashar, Lecky, Bryan R. F., Buckley, Camilla, Vincent, Angela, Dias-Tosta, Elza, Yoshikawa, Hiroaki, Waddington-Cruz, Márcia, Pulley, Michael T., Rivner, Michael H., Kostera-Pruszczyk, Anna, Pascuzzi, Robert M., Jackson, Carlayne E., Verschuuren, Jan J.G.M., Massey, Janice M., Kissel, John T., Werneck, Lineu C., Benatar, Michael, Barohn, Richard J., Tandan, Rup, Mozaffar, Tahseen, Silvestri, Nicholas J., Conwit, Robin, Sonett, Joshua R., Jaretzki, Alfred, Newsom-Davis, John, Cutter, Gary R.
Опубликовано в: Lancet Neurol (2019)