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Mavacamten Pregnancy Surveillance Program
The purpose of this observational pregnancy safety study is to assess maternal, fetal, and infant outcomes after exposure to mavacamten at any time during preg…
The EXerCise traIning To rEcovery in HCM Trial
The EXCITE-HCM study is a randomized, controlled, blinded clinical trial designed to evaluate the effect of moderate intensity exercise training versus usual p…
Prospective Study of the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders
Background: Pyrimidine and purine metabolism disorders (DPPMs) affect how the body metabolizes chemicals called pyrimidines and purines. DPPMs can cause dysfu…
A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study of Acoramidis for Transthyretin Amyloidosis Prevention in the Young (ACT-EARLY Trial)
Transthyretin amyloidosis (ATTR) is a disease where the normally occurring transthyretin (TTR) protein falls apart and forms amyloid, a sticky plaque- like sub…
A Prospective and Retrospective Registry and Biomarker Study to Evaluate the Natural History of Pediatric Patients With Cardiomyopathy Due to MYBPC3 Mutations
The objective of this study is to collect information on patients with cardiomyopathy (CM) due to mutations in the MYBPC3 gene, to evaluate their disease cours…
Smidt Heart Institute Takotsubo Registry
The Cedars-Sinai SHI Takotsubo Registry and Proteomic Study is an observational registry that will collect retrospective and prospective demographic, clinical,…
Rare Glycogen Storage Diseases Natural History Study
The purpose of this study is to collect and study key medical data about several ultra-rare GSDs (Glycogen Storage Diseases) including, but not limited to, GSD…
Randomized 3-Arm Trial With Standard of Care Alone vs Either Intravenous Infusion or Transendocardial Injection of Allogeneic Bone Marrow Derived Multipotent Mesenchymal Stromal Cells (MSCs) Plus Standard of Care in Patients With Anthracycline-Associated Cardiomyopathy
This randomized pilot phase I trial studies the side effects of donor bone marrow derived mesenchymal stem cells in controlling heart failure in patients with …
Implantable Cardioverter Defibrillator (ICD Registry)
The ICD Registry™ is a nationwide quality program that helps participating hospitals measure and improve care for patients receiving implantable cardioverter d…
Molecular Analysis of Neuromuscular Disease
In the Congenital Myopathy Research Program at Boston Children's Hospital and Harvard Medical School, the researchers are studying the congenital myopathies (n…
Dilated Cardiomyopathy Detection Using Artificial Intelligence and Screening With Mobile Technology
This is a prospective, single-arm clinical trial in which participants with dilated cardiomyopathy will invite their first degree relatives to undergo mobile c…
Real-World Camzyos Patient Reported OutcoMes Through PAtient SurveyS in Hypertrophic CardioMyopathy (COMPASS-HCM): A Prospective Longitudinal Study
This real-world study will assess changes in health status among participants with symptomatic obstructive hypertrophic cardiomyopathy who are treated with mav…