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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…
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…
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…
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…
A Phase 1 Dose Escalation Trial Evaluating an Intravenously Administered Recombinant Adeno-Associated Virus Serotype rh.74 (AAVrh.74) Vector Containing the Human Plakophilin-2a (PKP2a) Coding Sequence (RP-A601; AAVrh.74-PKP2a) in Subjects With Arrhythmogenic Cardiomyopathy Arising From Pathogenic PKP2 Variants (PKP2-ACM)
This Phase 1 dose escalation trial will assess the safety and preliminary efficacy of a single dose intravenous infusion of RP-A601 in high-risk adult patients…