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

por Waddington Cruz, Márcia, Benson, Merril D.
Publicado 2015

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

por Waddington Cruz, Márcia, Amass, Leslie, Keohane, Denis, Schwartz, Jeffrey, Li, Huihua, Gundapaneni, Balarama
Publicado 2016

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

por Merlini, Giampaolo, Coelho, Teresa, Waddington Cruz, Márcia, Li, Huihua, Stewart, Michelle, Ebede, Ben
Publicado 2020

Feasibility of assessing progression of transthyretin amyloid polyneuropathy using nerve conduction studies: Findings from the Transthyretin Amyloidosis Outcomes Survey (THAOS)

por Waddington‐Cruz, Márcia, Ando, Yukio, Amass, Leslie, Kiszko, Jan, Chapman, Doug, Sekijima, Yoshiki
Publicado 2021

Characteristics of Patients with Late- vs. Early-Onset Val30Met Transthyretin Amyloidosis from the Transthyretin Amyloidosis Outcomes Survey (THAOS)

por Waddington-Cruz, Márcia, Wixner, Jonas, Amass, Leslie, Kiszko, Jan, Chapman, Doug, Ando, Yukio
Publicado 2021

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

por Waddington-Cruz, Marcia, Amass, Leslie, Keohane, Denis, Schwartz, Jeffrey, Gundapaneni, Balarama, Li, Hui Hua
Publicado 2015

Optimal practices for the management of hereditary transthyretin amyloidosis: real-world experience from Japan, Brazil, and Portugal

por Ando, Yukio, Waddington-Cruz, Marcia, Sekijima, Yoshiki, Koike, Haruki, Ueda, Mitsuharu, Konishi, Hiroaki, Ishii, Tomonori, Coelho, Teresa
Publicado 2023

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

por Adams, David, Suhr, Ole B, Conceicao, Isabel, Waddington-Cruz, Marcia, Schmidt, Hartmut, Buades, Juan, Campistol, Josep, Coehlo, Teresa
Publicado 2015

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

por Waddington-Cruz, Márcia, Schmidt, Hartmut, Botteman, Marc F., Carter, John A., Stewart, Michelle, Hopps, Markay, Fallet, Shari, Amass, Leslie
Publicado 2019

Estimating the global prevalence of transthyretin familial amyloid polyneuropathy

por Schmidt, Hartmut H., Waddington‐Cruz, Márcia, Botteman, Marc F., Carter, John A., Chopra, Avijeet S., Hopps, Markay, Stewart, Michelle, Fallet, Shari, Amass, Leslie
Publicado 2018

Inflammatory profiling of patients with familial amyloid polyneuropathy

por Azevedo, Estefania P., Guimaraes-Costa, Anderson B., Bandeira-Melo, Christianne, Chimelli, Leila, Waddington-Cruz, Marcia, Saraiva, Elvira M., Palhano, Fernando L., Foguel, Debora
Publicado 2019

Early Data on Long-term Impact of Inotersen on Quality-of-Life in Patients with Hereditary Transthyretin Amyloidosis Polyneuropathy: Open-Label Extension of NEURO-TTR

por Yarlas, Aaron, Lovley, Andrew, McCausland, Kristen, Brown, Duncan, Vera-Llonch, Montserrat, Conceição, Isabel, Karam, Chafic, Khella, Sami, Obici, Laura, Waddington-Cruz, Márcia
Publicado 2021

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

por 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
Publicado 2015

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

por 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
Publicado 2022

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

por 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.
Publicado 2020

Temporal Trends of Wild-Type Transthyretin Amyloid Cardiomyopathy in the Transthyretin Amyloidosis Outcomes Survey

por Nativi-Nicolau, Jose, Siu, Alfonso, Dispenzieri, Angela, Maurer, Mathew S., Rapezzi, Claudio, Kristen, Arnt V., Garcia-Pavia, Pablo, LoRusso, Samantha, Waddington-Cruz, Márcia, Lairez, Olivier, Witteles, Ronald, Chapman, Doug, Amass, Leslie, Grogan, Martha
Publicado 2021

Liver‐directed drugs for transthyretin‐mediated amyloidosis

por Brannagan, Thomas H., Berk, John L., Gillmore, Julian D., Maurer, Mathew S., Waddington‐Cruz, Márcia, Fontana, Marianna, Masri, Ahmad, Obici, Laura, Brambatti, Michela, Baker, Brenda F., Hannan, Lisa A., Buchele, Gustavo, Viney, Nick J., Coelho, Teresa, Nativi‐Nicolau, Jose
Publicado 2022

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

por 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
Publicado 2020

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

por 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
Publicado 2019

Characteristics of Patients with Hereditary Transthyretin Amyloidosis-Polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an Open-label Phase 3 Study of Eplontersen

por Coelho, Teresa, Waddington Cruz, Márcia, Chao, Chi-Chao, Parman, Yeşim, Wixner, Jonas, Weiler, Markus, Barroso, Fabio A., Dasgupta, Noel R., Jung, Shiangtung W., Schneider, Eugene, Viney, Nicholas J., Dyck, P. James B., Ando, Yukio, Gillmore, Julian D., Khella, Sami, Gertz, Morie A., Obici, Laura, Berk, John L.
Publicado 2022