Cargando…

Liver‐directed drugs for transthyretin‐mediated amyloidosis

Transthyretin‐mediated amyloidosis (ATTR) is a rare, under‐recognized, progressively debilitating, fatal disease caused by the aggregation and extracellular deposition of amyloid transthyretin (TTR) fibrils in multiple organs and tissues throughout the body. TTR is predominantly synthesized by the l...

Descripción completa

Detalles Bibliográficos
Autores principales:	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
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Wiley Periodicals, Inc. 2022
Materias:	Reviews
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10100204/ https://www.ncbi.nlm.nih.gov/pubmed/36345805 http://dx.doi.org/10.1111/jns.12519

Ejemplares similares

Transthyretin cardiac amyloidosis
por: Porcari, Aldostefano, et al.
Publicado: (2022)

Temporal Trends of Wild-Type Transthyretin Amyloid Cardiomyopathy in the Transthyretin Amyloidosis Outcomes Survey
por: Nativi-Nicolau, Jose, et al.
Publicado: (2021)

Guideline of transthyretin-related hereditary amyloidosis for clinicians
por: Ando, Yukio, et al.
Publicado: (2013)

RNA Targeting and Gene Editing Strategies for Transthyretin Amyloidosis
por: Ioannou, Adam, et al.
Publicado: (2023)

Inotersen preserves or improves quality of life in hereditary transthyretin amyloidosis
por: Coelho, Teresa, et al.
Publicado: (2019)

Neuropathy symptom and change: Inotersen treatment of hereditary transthyretin amyloidosis
por: Dyck, P. James B., et al.
Publicado: (2020)

Characteristics of Patients with Hereditary Transthyretin Amyloidosis-Polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an Open-label Phase 3 Study of Eplontersen
por: Coelho, Teresa, et al.
Publicado: (2022)

Demyelinating Neuropathy in a Patient Treated With Revusiran for Transthyretin (Thr60Ala) Amyloidosis
por: Zanazzi, George, et al.
Publicado: (2019)

Avoiding misdiagnosis: expert consensus recommendations for the suspicion and diagnosis of transthyretin amyloidosis for the general practitioner
por: Gertz, Morie, et al.
Publicado: (2020)

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 Polyneuropathy
por: Coelho, Teresa, et al.
Publicado: (2021)

Disease progression in cardiac transthyretin amyloidosis is indicated by serial calculation of National Amyloidosis Centre transthyretin amyloidosis stage
por: Law, Steven, et al.
Publicado: (2020)

A Review of Tafamidis for the Treatment of Transthyretin-Related Amyloidosis
por: Waddington Cruz, Márcia, et al.
Publicado: (2015)

Impact of Vutrisiran on Quality of Life and Physical Function in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy
por: Obici, Laura, et al.
Publicado: (2023)

Expert opinion on monitoring symptomatic hereditary transthyretin-mediated amyloidosis and assessment of disease progression
por: Adams, David, et al.
Publicado: (2021)

Tafamidis for autonomic neuropathy in hereditary transthyretin (ATTR) amyloidosis: a review
por: Cruz, Márcia Waddington
Publicado: (2019)

Transthyretin cardiac amyloidosis in continental Western Europe: an insight through the Transthyretin Amyloidosis Outcomes Survey (THAOS)
por: Damy, Thibaud, et al.
Publicado: (2019)

Characteristics of Patients with Late- vs. Early-Onset Val30Met Transthyretin Amyloidosis from the Transthyretin Amyloidosis Outcomes Survey (THAOS)
por: Waddington-Cruz, Márcia, et al.
Publicado: (2021)

Early-Onset of Transthyretin Amyloidosis in a Young Afro-Caribbean Woman With Thr60Ala Mutation
por: Keppel, Stormy C., et al.
Publicado: (2020)

Screening for ATTR amyloidosis in the clinic: overlapping disorders, misdiagnosis, and multiorgan awareness
por: Nativi-Nicolau, Jose N., et al.
Publicado: (2021)

Clinical and genetic profile of patients enrolled in the Transthyretin Amyloidosis Outcomes Survey (THAOS): 14-year update
por: Dispenzieri, Angela, et al.
Publicado: (2022)

ATTR amyloidosis during the COVID-19 pandemic: insights from a global medical roundtable
por: Brannagan, Thomas H., et al.
Publicado: (2021)

Impact of genotype and phenotype on cardiac biomarkers in patients with transthyretin amyloidosis – Report from the Transthyretin Amyloidosis Outcome Survey (THAOS)
por: Kristen, Arnt V., et al.
Publicado: (2017)

Misdiagnoses of transthyretin amyloidosis: a clinical and electrodiagnostic study
por: Cortese, Andrea, et al.
Publicado: (2015)

Recommendations for presymptomatic genetic testing and management of individuals at risk for hereditary transthyretin amyloidosis
por: Obici, Laura, et al.
Publicado: (2016)

Sex Differences in Wild-Type Transthyretin Amyloidosis: An Analysis from the Transthyretin Amyloidosis Outcomes Survey (THAOS)
por: Campbell, Courtney M., et al.
Publicado: (2022)

Burden of hereditary transthyretin amyloidosis on quality of life
por: Yarlas, Aaron, et al.
Publicado: (2019)

World Heart Federation Consensus on Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)
por: Brito, Dulce, et al.
Publicado: (2023)

Treatment of acquired transthyretin amyloidosis in domino liver transplantation
por: Tsamis, Konstantinos I., et al.
Publicado: (2022)

Optimal practices for the management of hereditary transthyretin amyloidosis: real-world experience from Japan, Brazil, and Portugal
por: Ando, Yukio, et al.
Publicado: (2023)

Prognosis of Transthyretin Cardiac Amyloidosis Without Heart Failure Symptoms
por: Gonzalez-Lopez, Esther, et al.
Publicado: (2022)

Characteristics and natural history of early-stage cardiac transthyretin amyloidosis
por: Law, Steven, et al.
Publicado: (2022)

Ligand conjugated antisense oligonucleotide for the treatment of transthyretin amyloidosis: preclinical and phase 1 data
por: Viney, Nicholas J., et al.
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, et al.
Publicado: (2021)

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, et al.
Publicado: (2021)

Long-term efficacy and safety of inotersen for hereditary transthyretin amyloidosis: NEURO-TTR open-label extension 3-year update
por: Brannagan, Thomas H., et al.
Publicado: (2022)

Effects of Tafamidis on Transthyretin Stabilization and Clinical Outcomes in Patients with Non-Val30Met Transthyretin Amyloidosis
por: Merlini, Giampaolo, et al.
Publicado: (2013)

Analysis of disease progression in patients with transthyretin cardiac amyloidosis
por: Gilmore, Julian D, et al.
Publicado: (2015)

Phase 2, open-label extension (OLE) study of revusiran, an investigational RNAi therapeutic for the treatment of patients with transthyretin cardiac amyloidosis
por: Gillmore, Julian D, et al.
Publicado: (2015)

Mechanism of Action and Clinical Application of Tafamidis in Hereditary Transthyretin Amyloidosis
por: Coelho, Teresa, et al.
Publicado: (2016)

Clinical Importance of Left Atrial Infiltration in Cardiac Transthyretin Amyloidosis
por: Bandera, Francesco, et al.
Publicado: (2022)

Cannot write session to /tmp/vufind_sessions/sess_gafhbo8hrimol7d73jc57muu11