Results 61 to 70 of about 31,379 (232)

Cardiac transplantation in transthyretin amyloid cardiomyopathy: Outcomes from three decades of tertiary center experience

Frontiers in Cardiovascular Medicine, 2023
AimsTransthyretin cardiac amyloidosis (ATTR-CM) is a progressive and fatal cardiomyopathy. Treatment options in patients with advanced ATTR-CM are limited to cardiac transplantation (CT).
Yousuf Razvi, Aldostefano Porcari, Aldostefano Porcari, Concetta Di Nora, Rishi K. Patel, Adam Ioannou, Muhammad U. Rauf, Ambra Masi, Steven Law, Liza Chacko, Tamer Rezk, Sriram Ravichandran, Janet Gilbertson, Dorota Rowczenio, Iona J. Blakeney, Nandita Kaza, David F. Hutt, Helen Lachmann, Ashutosh Wechalekar, William Moody, Sern Lim, Colin Chue, Carol Whelan, Lucia Venneri, Ana Martinez-Naharro, Marco Merlo, Gianfranco Sinagra, Ugolino Livi, Philip Hawkins, Marianna Fontana, Julian D. Gillmore   +30 more
doaj   +1 more source

Primary Bladder Amyloidosis Mimicking Bladder Cancer: A Rare Case Report [PDF]

Bladder amyloidosis is a rare condition that can mimic malignancy clinically and radiologically. A 44-year-old female patient was admitted to our clinic with macroscopic hematuria and dysuria.
BOZKURT, OZAN, Volkan Şen, SİNİRSİZ, CAN, Can Sinirsiz, TUNA, EMİNE BURÇİN, ŞEN, VOLKAN, Emine Burçin Tuna, Ozan Bozkurt   +7 more
core   +1 more source

Outcomes of Patients With AL Amyloidosis and End‐Stage Renal Disease After Initiation of Therapy

American Journal of Hematology, EarlyView.
ABSTRACT In AL amyloidosis, advanced renal disease can impact treatment options and cause significant morbidity. Patients with ESRD requiring dialysis are a group with currently unmet needs. In this collaborative study, we identified 362 patients from three European centers who initiated dialysis during the course of the disease.
Foteini Theodorakakou, Lilli Sophie Sester, Ute Hegenbart, Paolo Milani, Claudia Bellofiore, Erasmia Psimenou, Charikleia Gakiopoulou, Meletios A. Dimopoulos, Giovanni Palladini, Stefan O. Schönland, Efstathios Kastritis   +10 more
wiley   +1 more source

Exome Sequencing Uncovers Phenotypic and Genotypic Heterogeneity in 196 Indian Families Evaluated for Autoinflammatory Disorders

American Journal of Medical Genetics Part A, EarlyView.
ABSTRACT Autoinflammatory disorders (AIDs) are a clinically heterogeneous group of inborn errors of immunity primarily caused by dysregulation in the innate immune system. Clinical diagnosis is often challenging due to clinical heterogeneity and the overlapping phenotypes with other inborn errors of immunity and monogenic conditions that mimic AIDs ...
Vaishnavi Ashok Badiger, Periyasamy Radhakrishnan, Suma Balan, Anand P. Rao, Prajna Udupa, Karthik Vijay Nair, Bhagesh Hunakunti, Adarsh Pooradan Prasannakumar, Sanket Limaye, Huzail Gani Shaikh, Rohit Naik, Mahesh Janarthanan, Sudharshan Sridharan, Vishal Sondhi, Sheela Nampoothiri, Hitesh Shah, Sabrinath Mahadevan, Mahabaleshwar Mamadapur, Devang Pandya, Katta Mohan Girisha, Abhay Shivpuri, Amita Moirangthem, Jyothi Raghuram, Gandham Sri Lakshmi Bhavani, Sachin Jeevanagi, Girish Subramaniam, Amita Aggarwal, Anju Shukla, Dhanya Lakshmi Narayanan   +28 more
wiley   +1 more source

Decoding RNA regulation: Challenges and opportunities for RNA‐based therapies in Europe

British Journal of Clinical Pharmacology, EarlyView.
Abstract RNA‐based medicinal products represent a promising frontier in personalised medicine, offering sequence‐specific disease targeting at various molecular levels, yet their clinical translation in the European Union (EU) may be hindered by regulatory uncertainty around definitions and evidence requirements; this study therefore aims to identify ...
Olivia C. Lewis, Christine Leopold, Joyce M. Hoek, Raymond M. Schiffelers, Marie L. De Bruin   +4 more
wiley   +1 more source

Regulatory harmonization: Evolution, globalization and future directions

British Journal of Clinical Pharmacology, EarlyView.
Regulatory harmonization has become an increasingly important and accepted approach to streamline regulatory review processes and expedite access to safe, effective and high‐quality medicines globally. This review explores the evolution and current status of regulatory harmonization, convergence and reliance initiatives.
Orin Chisholm, Elaine Y. L. Blair, Christine Y. Lu   +2 more
wiley   +1 more source

Avian amyloidosis [PDF]

Avian Pathology, 1998
Although amyloid deposits have been described for more than a century and a half, its proteinaceous and fibrillar nature was not revealed until after 1950. Biochemical characterization of amyloids has brought to light that several non-related proteins can re-organize into amyloid fibrils. In some domestic and caged wild birds, and especially waterfowl,
Landman, W.J.M., Gruys, E., Gielkens, A.L.J.   +2 more
openaire   +3 more sources

IN SILICO STUDIES OF AMYLOID FORMATION AND AMYLOID STABILITY [PDF]

, 2020
The term “Amyloid” describes the precursor proteins misfolded and aggregated into fibril-like structures that are built by cross β-sheet subunit. The disease caused by depositing amyloid fibril in tissues and organs is called amyloidosis. Amyloidosis can
Wang, Wenhua
core  

Safety outcomes of antidiabetic medications: A comprehensive review of the EU summaries of product characteristics and international clinical practice guidelines

British Journal of Clinical Pharmacology, EarlyView.
Aims Information on the safety profile of antidiabetic medications is essential for informed treatment decisions in type 2 diabetes mellitus. Although this information is available in regulatory documents of individual drugs, a comprehensive overview across all approved antidiabetics is lacking.
David Liang, Daniala L. Weir, Safana Thevasahayam, Vera H. M. Deneer, Arno Siebes, Helga Gardarsdottir   +5 more
wiley   +1 more source

Prognostic mortality factors in advanced light chain cardiac amyloidosis: A prospective cohort study

ESC Heart Failure
Aims Predicting mortality in severe AL cardiac amyloidosis is challenging due to elevated biomarker levels and limited thresholds for stratifying severe cardiac damage.
Amira Zaroui, Mounira Kharoubi, Romain Gounot, Silvia Oghina, Charlotte Degoutte, Melanie Bezard, Arnault Galat, Soulef Guendouz, Louise Roulin, Vincent Audard, Vincent Leroy, Emmanuel Teiger, Elsa Poullot, Valérie Molinier‐Frenkel, Fabien Le Bras, Karim Belhadj, Jean‐Philippe Bastard, Soraya Fellahi, Jason Shourick, Francois Lemonier, Thibaud Damy   +20 more
doaj   +1 more source