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Browsing by Author "Waddington Cruz, Marcia"

Browsing by Author "Waddington Cruz, Marcia"

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  • A 15-year consolidated overview of data in over 6000 patients from the Transthyretin Amyloidosis Outcomes Survey (THAOS) 
    Gentile, Luca; Coelho, Teresa; Dispenzieri, Angela; Conceição, Isabel; Waddington Cruz, Marcia; Kristen, Arnt V.; Wixner, Jonas; Diemberger, Igor; Gonzalez-Moreno, Juan; Cariou, Eve; Maurer, Mathew S.; Planté-Bordeneuve, Violaine; Garcia-Pavia, Pablo; Tournev, Ivailo; Gonzalez-Costello, Jose; Gonzalez Duarte, Alejandra; Grogan, Martha; Mazzeo, Anna; THAOS investigators; Barroso, Fabio Adrián (BioMed Central, 2023-11-10)
    Background: Transthyretin amyloidosis (ATTR amyloidosis) is a progressive, multisystemic, life-threatening disease resulting from the deposition of variant or wild-type (ATTRwt amyloidosis) transthyretin amyloid fibrils ...
  • Characteristics of patients with autonomic dysfunction in the Transthyretin Amyloidosis Outcomes Survey (THAOS) 
    Barroso, Fabio Adrián; Coelho, Teresa; Dispenzieri, Angela; Conceição, Isabel; Waddington Cruz, Marcia; Wixner, Jonas; Maurer, Mathew S.; Rapezzi, Claudio; Planté-Bordeneuve, Violaine; Kristen, Arnt V.; González-Duarte, Alejandra; Chapman, Doug; Stewart, Michelle; Amass, Leslie; THAOS investigators (Taylor & Francis, 2022-09)
    Background: Autonomic dysfunction is common in transthyretin amyloidosis (ATTR amyloidosis), but its frequency, characteristics, and quality-of-life (QoL) impact are not well understood. Methods: The Transthyretin ...
  • Characteristics of Patients with Hereditary Transthyretin Amyloidosis-Polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an Open-label Phase 3 Study of Eplontersen 
    Coelho, Teresa; Waddington Cruz, Marcia; Chao, Chi-Chao; Parman, Yeşim; Wixner, Jonas; Weiler, Markus; Barroso, Fabio Adrián; Dasgupta, Noel R.; Jung, Shiangtung W.; Schneider, Eugene; Viney, Nicholas J.; Dyck, P. James B.; Ando, Yukio; Gillmore, Julian D.; Khella, Sami; Gertz, Morie; Obici, Laura; Berk, John L. (Springer, 2022-12-16)
    Introduction: Hereditary transthyretin (ATTRv) amyloidosis is a rare, severe, progressive, debilitating, and ultimately fatal disease caused by systemic deposition of transthyretin (TTR) amyloid fibrils. ATTRv amyloidosis ...
  • Characteristics of Patients with Late- vs. Early-Onset Val30Met Transthyretin Amyloidosis from the Transthyretin Amyloidosis Outcomes Survey (THAOS) 
    Waddington Cruz, Marcia; Wixner, Jonas; Amass, Leslie; Kiszko, Jan; Chapman, Doug; Ando, Yukio; THAOS investigators (Springer, 2021-05-22)
    Introduction: Hereditary transthyretin amyloidosis (ATTRv amyloidosis) is a clinically heterogeneous disease caused by mutations in the transthyretin (TTR) gene. The most common mutation, Val30Met, can manifest as an early- ...
  • Clinical and genetic profile of patients enrolled in the Transthyretin Amyloidosis Outcomes Survey (THAOS): 14-year update 
    Dispenzieri, Angela; Coelho, Teresa; Conceição, Isabel; Waddington Cruz, Marcia; Wixner, Jonas; Kristen, Arnt V.; Rapezzi, Claudio; Planté-Bordeneuve, Violaine; Gonzalez-Moreno, Juan; Maurer, Mathew S.; Grogan, Martha; Chapman, Doug; Amass, Leslie; Barroso, Fabio Adrián; THAOS investigators (BioMed Central, 2022-06-18)
    Background: Transthyretin amyloidosis (ATTR amyloidosis) is a rare, life-threatening disease caused by the accumulation of variant or wild-type (ATTRwt amyloidosis) transthyretin amyloid fibrils in the heart, peripheral ...
  • Clinical and Genotype Characteristics and Symptom Migration in Patients With Mixed Phenotype Transthyretin Amyloidosis from the Transthyretin Amyloidosis Outcomes Survey 
    Gonzalez-Moreno, Juan; Dispenzieri, Angela; Grogan, Martha; Coelho, Teresa; Tournev, Ivailo; Waddington Cruz, Marcia; Wixner, Jonas; Diemberger, Igor; Garcia-Pavia, Pablo; Chapman, Doug; Gupta, Pritam; Glass, Oliver; Amass, Leslie; THAOS investigators; Barroso, Fabio Adrián (Springer, 2023-12-20)
    Introduction: Transthyretin amyloidosis (ATTR amyloidosis) is primarily associated with a cardiac or neurologic phenotype, but a mixed phenotype is increasingly described. Methods: This study describes the mixed phenotype ...
  • 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, Thomas H.; Wang, Annabel K.; Coelho, Teresa; Waddington Cruz, Marcia; Polydefkis, Michael J.; Dyck, Peter J.; Plante-Bordeneuve, Violaine; Berk, John L.; Barroso, Fabio Adrián; Merlini, Giampaolo; Conceição, Isabel; Hughes, Steven G.; Kwoh, Jesse; Jung, Shiangtung W.; Guthrie, Spencer; Pollock, Michael; Benson, Merrill D.; Gertz, Morie; NEURO-TTR open-label extension investigators (Wiley, 2020-08)
    Background and purpose: Hereditary transthyretin (hATTR) amyloidosis causes progressive polyneuropathy resulting from transthyretin (TTR) amyloid deposition throughout the body, including the peripheral nerves. The efficacy ...
  • Eplontersen in Hereditary ATTR-polyneuropathy: Week 66 Final Analysis of the Phase 3 NEURO-TTRansform Study 
    Khella, Sami; Marques, Wilson; Chao, Chi-Chao; Yesim Parman, Fatma; Franca, Marcondes C.; Guo, Yuh-Cherng; Ro, Long-Sun; Calandra, Cristian; Kowacs, Pedro A.; Berk, John L.; Piera Obici, Laura; Barroso, Fabio Adrián; Conceição, Isabel; Schneider, Eugene; Viney, Nicholas J.; Dyck, P. James B.; Waddington Cruz, Marcia; Coelho, Teresa (American Academy of Neurology, 2023-04-24)
    Background ATTRv-PN is a rare, progressive, and debilitating disease caused by accumulation of amyloid fibrils composed of transthyretin (TTR) protein in multiple organ systems. Eplontersen, a ligand-conjugated antisense ...
  • Long-Term Efficacy and Safety of Inotersen for Hereditary Transthyretin Amyloidosis: NEURO-TTR Open-Label Extension 2-Year Update (S27.008) 
    Brannagan, Thomas H.; Waddington Cruz, Marcia; Wang, Annabel K.; Polydefkis, Michael J.; Dyck, Peter J.; Khella, Sami; Plante-Bordeneuve, Violaine; Berk, John L.; Barroso, Fabio Adrián; Merlini, Giampaolo; Conceição, Isabel; Hughes, Steven G.; Kwoh, Jesse; Jung, Shiangtung W.; Guthrie, Spencer; Pollock, Michael; Benson, Merrill D.; Gertz, Morie; Coelho, Teresa (Lippincott Williams & Wilkins, 2019-03-09)
    Objective: To provide an update on the long-term efficacy and safety of inotersen, an antisense oligonucleotide inhibitor of transthyretin protein production, in patients with hereditary transthyretin amyloidosis (hATTR) ...
  • Phenotypic characteristics of F64L, I68L, I107V, and S77Y ATTRv genotypes from the Transthyretin Amyloidosis Outcomes Survey (THAOS) 
    Gentile, Luca; Diemberger, Igor; Plante-Bordeneuve, Violaine; Mazzeo, Anna; Dori, Amir; Luigetti, Marco; Di Paolantonio, Andrea; Dispenzieri, Angela; Grogan, Martha; Waddington Cruz, Marcia; Adams, David; Inamo, Jocelyn; Kristen, Arnt V.; Cirami, Calogero Lino; Chapman, Doug; Gupta, Pritam; Glass, Oliver; Amass, Leslie; Barroso, Fabio Adrián (Public Library of Science, 2024-01-19)
    Transthyretin amyloidosis (ATTR amyloidosis) is a progressive, multi-systemic disease with wild-type (ATTRwt) and hereditary (ATTRv) forms. Over 130 variants associated with ATTRv amyloidosis have been identified, although ...

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