Results 51 to 60 of about 99,289 (260)

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

Erythropoietin in Familial Amyloidosis ATTR V30M

, 2013
Familial amyloidosis ATTR V30M is an hereditary disorder, the most frequent type of transthyretin related amyloidosis. The main manifestation of the disease is a sensory-motor and autonomic polyneuropathy.
Costa, P.P., Beirão, I.
core   +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

Evaluation of a new ELISA assay for monoclonal free‐light chain detection in patients with cardiac amyloidosis

eJHaem, 2022
The causal protein of amyloid light‐chain (AL) amyloidosis is a monoclonal immunoglobulin free light chain (mFLC), which must be quantified in the serum for patient diagnosis and monitoring.
Hajer Abroud, Asma Beldi‐Ferchiou, Vincent Audard, François Lemonnier, Fabien Le Bras, Karim Belhadj, Anissa Moktefi, Elsa Poullot, Khalil El Karoui, Jehan Dupuis, Alizée Maarek, Louise Roulin, Marie‐Hélène Delfau‐Larue, Silvia Oghina, Mounira Kharoubi, Mélanie Bézard, Amira Zaroui, Thibaud Damy, Valérie Molinier‐Frenkel   +18 more
doaj   +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

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

DNA Nanotechnology Meets Peptide and Protein Self‐Assembly

Chemistry – A European Journal, EarlyView.
Combining DNA nanotechnology with peptide and protein assembly provides complementary platforms for the rational engineering of functional biomaterials. This Perspective discusses the emerging field of self‐assembling DNA‐peptide and DNA‐protein hybrid systems that combine the structural precision and programmability of DNA nanotechnology with the ...
Marcel Hanke, Nanjundan Raghul, Adrian Keller, Veikko Linko, Verónica I. Dodero   +4 more
wiley   +1 more source

Primary amyloidosis with initial gastrointestinal manifestation. A case report [PDF]

, 2018
Introduction. Amyloidosis is a rare disease associated with extracellular accumulation of abnormal protein – amyloid in various organs and systems. This disease can be either acquired or hereditary, systemic or localized.
Sergey D. ILIEV, Savelina L. POPOVSKA, Tatyana M. BETOVA, Radoslav G. TRIFONOV, Paulina T. VLADOVA   +4 more
core   +2 more sources

Progression and prognostic significance of electrocardiographic findings in patients with cardiac amyloidosis

ESC Heart Failure, Volume 12, Issue 2, Page 809-818, April 2025.
Abstract Aims This study aimed to evaluate the change of the main electrocardiographic (ECG) characteristics and their prognostic role across the main subtypes of cardiac amyloidosis [light‐chain amyloidosis (AL) and hereditary (ATTRv) and wild‐type transthyretin amyloidosis (ATTRwt)].
Alessia Argirò, Mattia Zampieri, Carlotta Mazzoni, Carlo Fumagalli, Michela Baccini, Alessandra Mattei, Alberto Cipriani, Laura De Michieli, Aldostefano Porcari, Gianfranco Sinagra, Marco Merlo, Giacomo Tini, Beatrice Musumeci, Domitilla Russo, Pier Filippo Vianello, Marco Canepa, Roberto Licordari, Gianluca di Bella, Claudio Rapezzi, Federico Perfetto, Francesco Cappelli   +20 more
wiley   +1 more source