Results 111 to 120 of about 16,120 (219)

Small molecule amyloid disrupters demonstrate therapeutic efficacy for transthyretin amyloidosis

The escalating global trend of an aging population has brought attention to the rising prevalence of late-onset amyloid disorders. Among them, transthyretin amyloidosis (ATTR) presents a growing medical challenge, particularly in the elderly.
Genki, Kudo, Youhei, Sohma, Moe, Toyobe, Yukiko, Hori, Mina, Yamane, Shigehiro A., Kawashima, Hiroki, Umeda, Yukio, Ando, Masamitsu, Okada, Motomu, Kanai, Takatsugu, Hirokawa, Atsushi, Iwai, Mitsuharu, Ueda, Taisuke, Tomita, Kunitoshi, Yamanaka, Harunobu, Mitsunuma, Mineyuki, Mizuguchi   +16 more
core   +1 more source

Combination surgery for pressure control in glaucoma from transthyretin amyloidosis, 3-year follow-up

Indian Journal of Ophthalmology. Case Reports
Transthyretin amyloidosis is a known cause of secondary glaucoma. The authors present a 3-year update on a previously published patient who underwent Kahook Dual Blade (KDB) excisional goniotomy in the right eye, followed by KDB combined with cataract ...
Natalie Brossard-Barbosa, Valentina Campisi, Naomi E Gutkind, Carla Salina Indovino, Uriel Rubin, Tomas M Grippo   +5 more
doaj   +1 more source

Polyneuropathy in hereditary and wildtype transthyretin amyloidosis, comparison of key clinical features and red flags

Scientific Reports
Transthyretin (TTR) amyloidosis manifests in two distinct forms: hereditary (ATTRv) and wild-type transthyretin amyloidosis (ATTRwt). Despite being one of the commonest manifestations in ATTRv amyloidosis, the presence of polyneuropathy has long been ...
Janna M. Siemer, Lea Grote-Levi, Anja Hänselmann, Mieke L. Sassmann, Sandra Nay, Dominica Ratuszny, Sonja Körner, Tabea Seeliger, Martin W. Hümmert, Maike F. Dohrn, André Huss, Hayrettin Tumani, Vega Gödecke, Michael Heuser, Johann Bauersachs, Udo Bavendiek, Thomas Skripuletz, Stefan Gingele   +17 more
doaj   +1 more source

Pompe Disease Could Mimic Exam Findings of Amyloidosis: Two Rare Diagnoses Bona Fide

Case Reports in Hematology, 2018
A 70-year-old female presented with a three-year history of evolving macroglossia causing dysphagia and dysarthria, with proximal muscle weakness.
Jithma P. Abeykoon, Narjust Duma, Jennifer A. Tracy, Margherita Milone, Ronald Go   +4 more
doaj   +1 more source

What's new in the treatment of amyloidosis? Part 2: Cardiomyopathy due to transthyretin amyloidosis

Revista del Hospital Italiano de Buenos Aires
Transthyretin deposition amyloidosis is a rare disease caused by the deposition of fibrils of this protein in various tissues, although the most common manifestations are cardiac and neurological.
Gisela Bendelman, Marcelina Carretero, Diego Pérez de Arenaza, Eugenia Villanueva, Erika B. Brulc, Elsa M. Nucifora, María A. Marco, María S. Sáez, Patricia Sorroche, María A. Aguirre, María L. Posadas Martínez   +10 more
doaj   +1 more source

Tafamidis Reduces Death and Hospitalization for Acute Heart Failure in Octogenarian Patients With Transthyretin Cardiac Amyloidosis: A Propensity Score–Weighted Cohort Study

Journal of the American Heart Association: Cardiovascular and Cerebrovascular Disease
Background Transthyretin cardiac amyloidosis results from the deposition of misfolded amyloid fibrils in cardiac tissue, leading to progressive heart failure.
Louise Zmuda, Amira Zaroui, Mounira Kharoubi, Emmanuelle Boutin, Frédéric Roca, Silvia Oghina, Emmanuel Teiger, Marie Laurent, Florence Canoui‐Poitrine, Thibaud Damy, Amaury Broussier   +10 more
doaj   +1 more source

Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy

New England Journal of Medicine
Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) is a progressive, fatal disease. Vutrisiran, a subcutaneously administered RNA interference therapeutic agent, inhibits the production of hepatic transthyretin.In this double-blind, randomized trial, we assigned patients with ATTR-CM in a 1:1 ratio to receive vutrisiran (25 mg) or placebo every 12
Marianna Fontana, John L. Berk, Julian D. Gillmore, Ronald M. Witteles, Martha Grogan, Brian Drachman, Thibaud Damy, Pablo Garcia-Pavia, Jorg Taubel, Scott D. Solomon, Farooq H. Sheikh, Nobuhiro Tahara, José González-Costello, Kenichi Tsujita, Caroline Morbach, Zoltán Pozsonyi, Mark C. Petrie, Diego Delgado, Peter Van der Meer, Andrew Jabbour, Antoine Bondue, Darae Kim, Olga Azevedo, Steen Hvitfeldt Poulsen, Ali Yilmaz, Ewa A. Jankowska, Vincent Algalarrondo, Andrew Slugg, Pushkal P. Garg, Katherine L. Boyle, Elena Yureneva, Nancy Silliman, Lilli Yang, Jihong Chen, Satish A. Eraly, John Vest, Mathew S. Maurer   +36 more
openaire   +4 more sources

Cardiac Amyloidosis: A Narrative Review of Diagnostic Advances and Emerging Therapies

Biomedicines
Background/Objectives: Cardiac amyloidosis (CA) is an underdiagnosed and potentially life-threatening infiltrative cardiomyopathy characterized by the extracellular deposition of misfolded amyloid fibrils in cardiac tissue. It is most commonly associated
Dana Emilia Movila, Alexandru Catalin Motofelea, Dragos Cozma, Oana Albai, Alexandra Christa Sima, Minodora Andor, Tudor Ciocarlie, Simona Ruxanda Dragan   +7 more
doaj   +1 more source

Diagnosis and Screening of Patients with Hereditary Transthyretin Amyloidosis (hATTR): Current Strategies and Guidelines

, 2020
MD Benson,1 NR Dasgupta,1,2 R Rao2 1Department of Pathology & Laboratory Medicine, Indiana University School of Medicine, Indianapolis, IN, USA; 2Division of Cardiology, Indiana University School of Medicine, Indianapolis, IN 46040, USACorrespondence:
Rao R, Benson MD, Dasgupta NR
core  

Patient characteristics for men and women with transthyretin amyloidosis.

, 2015
Type A, mixture of intact and fragmented transthyretin; Type B, only full length transthyretin. Continuous data are presented as median (range) and categorical data are presented as counts and percentages. Statistically significant differences are marked
Per Westermark (79625), Björn Pilebro (829908), Per Lindqvist (829909), Ole B. Suhr (298443), Sandra Arvidsson (829907)   +4 more
core   +1 more source