Results 61 to 70 of about 2,198,265 (159)

Impact of Baseline Polyneuropathy Severity on Eplontersen Efficacy in the NEURO‐TTRansform Clinical Trial

Journal of the Peripheral Nervous System, Volume 31, Issue 3, September 2026.
ABSTRACT Background and Aims In the NEURO‐TTRansform clinical trial (NCT04136184), eplontersen improved neuropathy impairment and quality of life (QoL) through Week 66 versus the NEURO‐TTR historical placebo in patients with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv‐PN).
Thomas H. Brannagan III, Markus Weiler, Márcia Waddington Cruz, Sami Khella, John L. Berk, Morie Gertz, Ahmad Masri, Mathew S. Maurer, Jonas Wixner, Jonatan Nåtman, T. Jesse Kwoh, Maksym Pola, Laura Obici   +12 more
wiley   +1 more source

Proportion of ATTR-CM-reference and ATTR-CM-like cases in SUS according to the ML model classification from 2015 to 2021.

Proportion of ATTR-CM-reference and ATTR-CM-like cases in SUS according to the ML model classification from 2015 to 2021.
Fabio Fernandes (3386423), Isabella Zuppo Laper (17972784), Marcus Vinicius Simões (5224412), Claudenice Leite Bertoli de Souza (17972790), Ariane de Jesus Lopes de Abreu (14425084), Rafaela Vansan Ferreira (14425090), Edileide de Barros Correia (17972793), Guilherme Silva Julian (8520117), Cecilia Camacho-Hubner (17972787)   +8 more
core   +1 more source

Treatment patterns and healthcare resources utilization of ATTR-CM patients, from 2015 to 2021, per ATTR-CM type.

Treatment patterns and healthcare resources utilization of ATTR-CM patients, from 2015 to 2021, per ATTR-CM type.
Fabio Fernandes (3386423), Isabella Zuppo Laper (17972784), Marcus Vinicius Simões (5224412), Claudenice Leite Bertoli de Souza (17972790), Ariane de Jesus Lopes de Abreu (14425084), Rafaela Vansan Ferreira (14425090), Edileide de Barros Correia (17972793), Guilherme Silva Julian (8520117), Cecilia Camacho-Hubner (17972787)   +8 more
core   +1 more source

Suspicion and referral of patients with transthyretin amyloid cardiomyopathy: Recommendations by a Portuguese multidisciplinary expert panel

Revista Portuguesa de Cardiologia
Early diagnosis of transthyretin amyloid cardiomyopathy (ATTR-CM) is crucial for better disease management and outcome. To ensure timely diagnosis, a multidisciplinary panel of Portuguese experts, including cardiologists, internal medicine specialists ...
Dulce Brito, João Agostinho, Carlos Aguiar, Sílvia Aguiar Rosa, Nuno Cardim, Cândida Fonseca, Nuno Marques, Pedro Moraes Sarmento, Patrícia Rodrigues, Jonathan Santos, Maria João Vidigal Ferreira, Olga Azevedo   +11 more
doaj   +1 more source

DMS‐MaPseq and DREEM Analyses Implicate the Critical Role of RNA Structural Dynamics in Turnip Yellow Mosaic Virus Pathogenicity

Advanced Science, Volume 13, Issue 43, 3 August 2026.
RNA structural profiling of Turnip Yellow Mosaic Virus by DMS‐MaPseq and DREEM analyses uncover that viral genome‐wide RSS is highly complicated and heterogeneous, with alternative RSSs widely distributed across the genome. Notably, the viral 3’ tRNA‐like structure adopts alternative conformations in vivo.
Jiaying Zhu, Changhao Li, Chisimbily Onyia, Xindi Li, Xingxing Yan, Songxiao Zhong, Taerin Oh, Xiuren Zhang   +7 more
wiley   +1 more source

INSIGHT Model

JACC: Advances
Background: Transthyretin amyloidosis (ATTR) diagnoses are frequently delayed or missed, attenuating treatment benefit. Objectives: The authors developed an explainable artificial intelligence screening tool to identify high-risk patients who should be ...
Akshay Arora, MS, Muhammad Shahzeb Khan, MD, MSc, Cynthia Sunderman, MSN, Nicole Baldwin, PhD, MS, Monica Bennett, PhD, Vee Anne Bomar, Lisa Cross, PhD, MSc, Briget da Graca, JD, MS, Jason Ettlinger, MA, Robert L. Gottlieb, MD, PhD, Kendall Hammonds, MPH, Michael Mack, MD, Ahmad Masri, MD MS, Mia Papas, PhD, Elisa L. Priest, DrPH, MPH, Courtney Shaver, MS, John Venditto, MD, Javed Butler, MD, MPH, MBA   +17 more
doaj   +1 more source

Clinical Pharmacology of Eplontersen, the First Approved GalNAc‐Conjugated Antisense Oligonucleotide

The Journal of Clinical Pharmacology, Volume 66, Issue 8, August 2026.
Abstract Eplontersen is a GalNAc‐conjugated antisense oligonucleotide (ASO) approved for the treatment of hereditary transthyretin‐mediated amyloidosis with polyneuropathy (ATTRv‐PN). GalNAc conjugation enables targeted hepatic delivery and improved potency compared with unconjugated ASOs.
Rosie Z. Yu, John K. Diep, Xiang Gao, Cecilia Arfvidsson, Angelica Quartino   +4 more
wiley   +1 more source

Evolution of patient characteristics and outcomes in ATTR-CM clinical trials: A systematic review.

Background: Over the past decade, advancements in awareness, diagnosis, and new treatment options for transthyretin amyloid cardiomyopathy (ATTR-CM) have likely influenced the characteristics of patient populations enrolled in clinical trials.
Betts, Marissa, Nativi-Nicolau, Jose, Masri, Ahmad, Dev, Sandesh, Hennum, Liana, Wright, Richard, Falvey, Heather, Lavoie, Louis, Tamby, Jean-François   +8 more
core   +1 more source

Assessment of potential transthyretin amyloid cardiomyopathy cases in the Brazilian public health system using a machine learning model.

PLoS ONE
ObjectivesTo identify and describe the profile of potential transthyretin cardiac amyloidosis (ATTR-CM) cases in the Brazilian public health system (SUS), using a predictive machine learning (ML) model.MethodsThis was a retrospective descriptive database
Isabella Zuppo Laper, Cecilia Camacho-Hubner, Rafaela Vansan Ferreira, Claudenice Leite Bertoli de Souza, Marcus Vinicius Simões, Fabio Fernandes, Edileide de Barros Correia, Ariane de Jesus Lopes de Abreu, Guilherme Silva Julian   +8 more
doaj   +1 more source

Investigating transthyretin variants H88R and I107V in amyloid priming: From destabilization to complete dissociation

The FEBS Journal, Volume 293, Issue 15, Page 4680-4703, August 2026.
Investigated mutations in transthyretin (TTR) disrupt the F87‐centered hydrophobic core that stabilizes its tetrameric structure. The mild I107V mutation weakens inter‐chain packing, while H88R fully abolishes tetramer formation, yielding a monomeric, aggregation‐prone form. Structural, biophysical, and computational analyses reveal that both mutations
István L. Bódy, Zsolt Fazekas, Nóra Wágner, Anna J. Kiss‐Szemán, Veronika Harmat, Zoltán Pozsonyi, Dóra K. Menyhárd, András Perczel   +7 more
wiley   +1 more source