Results 61 to 70 of about 5,684 (155)
Anuja Roy,1 Andrew Peterson,2 Nick Marchant,1 Jose Alvir,3 Rahul Bhambri,4 Jason Lynn,4 Darrin Benjumea,5 Sapna Prasad,6 Alex O’Brien,6 Yong Chen,7 Jason Kemner,8 Bhash Parasuraman8 1Global HEOR, Patient & Health Impact, Rare Diseases BU, Pfizer Inc, New
Roy A +11 more
doaj
Histopathological examination of an abdominal fat pad core needle biopsy demonstrating Congo red–positive amyloid deposition, confirming systemic amyloidosis. Representative photomicrographs illustrate characteristic amyloid deposits at low‐ and high‐power magnification.
Malegna Temesgen Garuma +2 more
wiley +1 more source
Introduction Tafamidis is the first drug approved by the European Commission for the treatment of wild-type or hereditary transthyretin amyloid cardiomyopathy (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related ...
Sepideh Attal +4 more
doaj +1 more source
This exploratory analysis of data from the NEURO‐TTRansform trial evaluated sex‐specific responses in patients with ATTRv‐PN who had received eplontersen in NEURO‐TTRansform versus a historical placebo. Eplontersen can lead to substantial decreases in percentage serum TTR levels, arrest neuropathy impairment, and improve patient quality of life ...
Márcia Waddington Cruz +11 more
wiley +1 more source
This summary presents the results from an ongoing, long-term extension study that followed an earlier study called ATTR-ACT. People who took part in this extension study and ATTR-ACT have a type of heart disease known as transthyretin amyloid ...
Sultan, Marla B +21 more
core +1 more source
Early‐Onset Wild‐Type Transthyretin Amyloidosis Polyneuropathy
Muscle &Nerve, Volume 74, Issue 5, Page 1471-1475, November 2026.
Chafic Karam +7 more
wiley +1 more source
Introduction: Hereditary transthyretin amyloidosis (vATTR) is a progressive genetic disorder with several approved treatments. We investigated treatment responses to tafamidis and patisiran in vATTR patients to identify predictive response factors ...
L. Martínez-Vicente +14 more
doaj +1 more source
Benzofuran‐Based Uricosuric Agents: History, Roles, and Challenges
Benzofuran‐based uricosuric agents represent a privileged medicinal chemistry scaffold for gout therapy. Structural optimization of benzbromarone‐related derivatives has improved hURAT1 inhibitory potency, metabolic stability, and urate‐lowering efficacy, while reducing hepatotoxic liability.
Raed M. Al‐Zoubi +7 more
wiley +1 more source
Tafamidis: A First-in-Class Transthyretin Stabilizer for Transthyretin Amyloid Cardiomyopathy.
Objective: To review the pharmacology, efficacy, and safety of the selective transthyretin inhibitor tafamidis for transthyretin amyloid cardiomyopathy (ATTR-CM). Data Sources: A PubMed (1966 to October 2019) and ClinicalTrials.
Andrews, Ebony +5 more
core +1 more source
Catarina Falcão de Campos,1,2 Isabel Conceição1,2 1Department of Neurosciences and Mental Health, Centro Hospitalar Universitário de Lisboa-Norte, Lisbon, Portugal; 2Instituto de Fisiologia, Instituto de Medicina Molecular, Centro de Estudos Egas Moniz ...
Falcão de Campos C, Conceição I
doaj

