Characterization and Natural History of Patients with <i>LMNA</i> -Related Dilated Cardiomyopathy in the Phase 3 REALM-DCM Trial
Findings confirm the significant morbidity and mortality associated with LMNA-related DCM despite the standard of care management. Typical measures of HF, including 6MWT distance, KCCQ-OS score and NT-proBNP concentration, were variable but correlated with NYHA class. An unmet treatment need remains among patients with LMNA-related DCM. NCT03439514.
Authors
- Perry Elliott (ORCID: https://orcid.org/0000-0003-3383-3984)
- Silvia Giuliana Priori (ORCID: https://orcid.org/0000-0001-6877-0288)
- James S. Ware (ORCID: https://orcid.org/0000-0002-6110-5880)
- Gianfranco Sinagra (ORCID: https://orcid.org/0000-0003-2700-8478)
- ANJALI TIKU OWENS (ORCID: https://orcid.org/0000-0002-9669-8495)
- Neal K. Lakdawala (ORCID: https://orcid.org/0000-0001-6458-5421)
- Calum A. MacRae (ORCID: https://orcid.org/0000-0001-5181-2664)
- Pablo García‐Pavía (ORCID: https://orcid.org/0000-0002-5470-2257)
- Daniel Philip Judge (ORCID: https://orcid.org/0000-0002-3407-0248)
- Tomás Ripoll‐Vera (ORCID: https://orcid.org/0000-0001-9222-325X)
- Hui‐Hua Li (ORCID: https://orcid.org/0000-0001-5983-4016)
- Franca S. Angeli
- K. Afshar
Institutions
- Intermountain Healthcare (US)
- Brigham and Women's Hospital (US)
- Harvard University (US)
- University of Trieste (IT)
- Medical University of South Carolina (US)
- Pfizer (United States) (US)
- Guy's and St Thomas' NHS Foundation Trust (GB)
- Universidad Francisco de Vitoria (ES)
- University of Pavia (IT)
- Royal Brompton & Harefield NHS Foundation Trust (GB)
- Instituto de Salud Carlos III (ES)
- Spanish Biomedical Research Centre in Physiopathology of Obesity and Nutrition (ES)
- Harefield Hospital (GB)
- Intermountain Medical Center (US)
- Spanish National Centre for Cardiovascular Research (ES)
- Hospital Son Llatzer (ES)
- Istituti Clinici Scientifici Maugeri (IT)
- Lankenau Heart Institute (US)
- Centro de Investigación en Red en Enfermedades Cardiovasculares (ES)
- Lung Institute (US)
- Hospital Universitario Puerta de Hierro Majadahonda (ES)
- Health Research Institute of the Balearic Islands (ES)
- Instituto de Investigación Sanitaria de Santiago (ES)
- MRC London Institute of Medical Sciences (GB)
- Azienda Sanitaria Universitaria Integrata di Trieste (IT)
- University College London (GB)
- Imperial College London (GB)
- Universitat de les Illes Balears (ES)
- University of Pennsylvania (US)
Publication Details
- Journal
- ESC Heart Failure
- Published
- 2024-08-15
- DOI
- https://doi.org/10.1002/ehf2.14955
- Citations
- 4
- Primary Topic
- Nuclear Structure and Function
- Type
- article
- Field-Weighted Citation Impact
- 0.94
Funders
- Bristol-Myers Squibb
- Pfizer
- AstraZeneca
- Sanofi
- Alexion Pharmaceuticals
- Alnylam Pharmaceuticals
- Array BioPharma
- Boston Scientific Corporation
- Cytokinetics
- Sarepta Therapeutics
- Amicus Therapeutics
- MyoKardia
- Impulse Dynamics
- National Institute for Health and Care Research
- British Heart Foundation
- Novo Nordisk
- Vifor Pharma
- Medical Research Council
- NIHR Imperial Biomedical Research Centre