Results 101 to 110 of about 32,506 (196)

The features of cardiomyopathy in AL and ATTR amyloidosis

Rheumatology (Bulgaria)
Amyloidoses are protein misfolding diseases characterized by the accumulation of amyloid fibrils in the interstitium leading to the damage of an affected organ. Cardiac involvement is quite common and results from light chain amyloidosis (AL) or transthyretin amyloidosis (ATTR), including two subtypes: wild type (ATTRwt) and variant ATTR (ATTRv), which
Stefka Gospodinova Radenska-Lopovok, N. Y. Feyziyeva, K. R. Pashayeva, B. Ponomarev   +3 more
openaire   +1 more source

Generation of two induced pluripotent stem cell lines from hereditary amyloidosis patients with polyneuropathy carrying heterozygous transthyretin (TTR) mutation

Stem Cell Research
Hereditary transthyretin amyloidosis with polyneuropathy (ATTR-PN) results from specific TTR gene mutations. In this study, we generated two induced pluripotent stem cell (iPSC) lines derived from ATTR-PN patients with heterozygous TTR gene mutations ...
Juan Melesio, Bernardo Bonilauri, Audrey Li, Paul D. Pang, Ronglih Liao, Ronald M. Witteles, Joseph C. Wu, Karim Sallam   +7 more
doaj   +1 more source

Advancing Transthyretin Amyloidosis Drug Development in an Evolving Treatment Landscape: Amyloidosis Forum Meeting Proceedings [PDF]

INTRODUCTION: Hereditary transthyretin amyloidosis (ATTRv, also referred to as hATTR; ORPHA 271861) and wild-type ATTR amyloidosis (ATTRwt; ORPHA 330001) are rare, progressive, systemic protein misfolding disorders with heterogeneous clinical ...
Soman, Prem, Kittleson, Michelle, Hsu, Kristen, Hanna, Mazen, Lousada, Isabelle, Amyloidosis Forum Meeting Panelists, Adigun, Rosalyn, Dunnmon, Preston, Gillmore, Julian, Ruberg, Frederick L, Kelly, Jeffery, Hernandez, Adrian, Dorbala, Sharmila, Maurer, Mathew S, Signorovitch, James, Garcia-Pavia, Pablo, Wei, LJ, Kazi, Dhruv   +17 more
core   +3 more sources

Content Validation of the ATTR Amyloidosis Patient Symptom Survey: Findings from Patient and Clinician Cognitive Debriefing Interviews

Patient Related Outcome Measures, 2020
Avery A Rizio,1 Lynne E Broderick,1 Michelle K White,1 Tiffany P Quock2 1Optum Life Sciences, Patient Insights, Johnston, RI, USA; 2Prothena Biosciences Inc, South San Francisco, CA, USACorrespondence: Avery A RizioOptum Life Sciences, Patient Insights ...
Rizio AA, Broderick LE, White MK, Quock TP   +3 more
doaj  

Analysis of ex vivo ATTR fibrils from patients with systemic ATTR amyloidosis

Transthyretin amyloidosis is a rare and very diverse disease in which amyloid fibrils derived from transthyretin (TTR) deposit within the body. The deposition of amyloid transthyretin (ATTR), usually in the interstitial space between cells, can appear at
Steinebrei, Maximilian
core   +1 more source

Diagnostic performance of transthyretin measurement in fat tissue of patients with ATTR amyloidosis

, 2010
Background: The diagnostic performance was studied of a transthyretin (TTR) ELISA for detection and characterisation of transthyretin-derived (ATTR) amyloid in abdominal subcutaneous fat tissue.
Hazenberg, B.P.C., Haagsma, E.B., Bijzet, J., Bos, R., Van Schijndel, B., Hazenberg, B.P.C.; id_orcid, Limburg, P.C., Bijzet, J.; id_orcid   +7 more
core   +1 more source

Transferability of a US claims-based machine learning model for ATTRwt-CM identification: a retrospective evaluation in a German setting

Scientific Reports
Cardiac amyloidosis (CA), a fatal and progressive cardiomyopathy is characterized by amyloid deposition within the myocardium. The main forms of CA include transthyretin amyloidosis (ATTR-CM; distinguished into a hereditary and wildtype form [ATTRv and ...
Harisa Muratovic-Colic, Miriam R. Hübner, Jan-Filip Rehburg, Isabel Mattig, Paul J. Wetzel, Katrin Wrede-Wihl, Helena F. Pernice, Gina Barzen, Nicolas Wieder, Jakub Piwowarski, Stephan Bohl, Vera von Landenberg-Roberg, Daniel Messroghli, Sebastian Spethmann, Richard Röttger, Josef Schepers, Katrin Hahn   +16 more
doaj   +1 more source

Central Nervous System Involvement as the Initial Manifestation of Transthyretin Amyloidosis: A Case Report and Literature Review

iRADIOLOGY
Transthyretin amyloidosis (ATTR), a rare systemic disorder characterized by misfolded transthyretin (TTR) protein aggregation, predominantly affects the heart and peripheral nervous system.
Fen Li, Mingsheng Wan, Benjian Sun, Lijia Zou, Huan Yang, Jing Li, Si Chen   +6 more
doaj   +1 more source

Native T1 and ECV in ATTR amyloidosis [PDF]

, 2019
Objectives This study evaluated the prognostic potential of native myocardial T1 in cardiac transthyretin amyloidosis (ATTR) and compared native T1 with extracellular volume (ECV) in terms of diagnostic accuracy and prognosis.
Martinez-Naharro, A
core   +3 more sources

A case of pulmonary transthyretin amyloidosis with concurrent mycobacterial tuberculosis infection

Respirology Case Reports
Amyloidosis is a pathological deposition disease that causes a spectrum of organ dysfunction. Pulmonary involvement is generally associated with immunoglobulin light chain type (AL) amyloid. Transthyretin (ATTR) amyloid build up in the lung is thought to
Hanson Siu, Ari Mond, James Shaw, Ruth Chin, Patrick Hosking, Hari Wimaleswaran   +5 more
doaj   +1 more source