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Genetic Cardiomyopathy Registry (GCR) FAQs ORIGINAL

1. What is the purpose of the Genetic Cardiomyopathy Registry? The purpose of the Genetic Cardiomyopathy Registry is to bring the Cardiomyopathy community together and collect data. Some of the goals of the Genetic Cardiomyopathy Registry are: To describe the people who have Cardiomyopathy and to better understand the stages of the disease and the different ways the disease affects people. To do this, we will ask about diagnosis, treatment, medical history, social and economic environment, and treatment outcomes. To identify significant numbers of participants with genetic subtypes of cardiomyopathies. To guide participants on where and how to obtain genetic…

Current Clinical Trials & Research Studies

Overview Research studies and clinical trials are carefully designed to evaluate the safety and effectiveness of potential treatments. Gene therapy clinical trials for cardiomyopathy provide patients the possibility of access to an investigational treatment and the ability to contribute to the advancement of scientific research that may also help other individuals with the disease. To learn more about Gene Therapy Clinical Trials and how they work, click here. Current Genetic Cardiomyopathy Clinical Trials PKP2 Clinical Trial (Click to learn more) Lexeo’s HEROIC-PKP2 Clinical Trial – for eligible patients with confirmed ACM (also called ARVC) caused by the PKP2 genetic mutation.…

Upcoming Research Opportunity

A new research study is inviting children with heart failure related to Danon disease or another heart condition and/or their caregivers to take part in one 75-minute interview. The purpose of the upcoming study is to: Learn about the experiences of children with heart failure related to Danon disease or another heart condition, and Gather feedback on a health questionnaire for patients and caregivers of patients with heart failure related to Danon disease or another condition What is involved? Being in this study means taking part in one 75-minute interview (by telephone or computer). During the interview, a researcher will…

About Clinical Trials

What is a Clinical Trial? Gene therapy, a rapidly evolving field, holds the potential to revolutionize how we treat genetic diseases by tackling them at their root cause. But before any drug or gene therapy is approved for use in humans, it must undergo a rigorous testing process called a clinical trial. Clinical trials assess the safety and effectiveness of a drug or gene therapy. FDA scientists conduct thorough evaluations during clinical trials to determine whether the potential benefits of a drug or therapy outweigh the possible associated risks. This stringent assessment process ensures that the therapy adheres to rigorous…

Thank You for Joining the Advocacy Army!

Spread Our Message and Help Save Lives! Thank you for helping us spread the word. Together, we can make a difference. We will continue to add materials to this tool kit to help you tell our story and save lives. See below for general instructions about how to use the items in this Tool Kit. Please also check your email for a thank you letter with instructions for use of the Advocacy Army Tool Kit. (If you didn’t receive an email, please check your spam folder.) Specific instructions for the use of each kind of marketing piece are included within the…

Join the Fight

Join our Advocacy Army and Help Save Lives! Every day, people with cardiomyopathy are in a battle for their lives. The Genetic Cardiomyopathy Awareness Consortium is on a mission to increase awareness and to get more cardiomyopathy patients and family members genetically tested, which will help save lives and advance genetic research and therapies. By becoming an advocate for Genetic Cardiomyopathy, you can help us reach more people, share important information, and bring us closer to a future free from this devastating disease. Please join our team by sharing the information we have provided in our Advocacy Army Tool Kit.…

Genetics 101

Let’s talk about GENES What are they and what do they do? The human body is made of trillions of tiny building blocks called cells. Almost all cells contain a nucleus, which is the control center of the cell. The nucleus contains our genetic material, called deoxyribonucleic acid or DNA, which is organized into pairs of thread-like structures called chromosomes. Passed from parents to children, DNA contains the specific instructions that make each person unique. A gene is usually a short section of DNA that contains instructions for making a specific protein or set of proteins Proteins are the molecules…

Gene Therapy Basics

Getting to the Root Cause of Cardiomyopathy Gene therapy holds the potential to treat the root cause of cardiomyopathy by targeting the genes that can lead to the condition. The future of gene therapy offers hope for those affected by cardiomyopathy, beyond just symptom management. What is Gene Therapy? Gene therapy is a way of treating or preventing conditions caused by genetic mutations. Think of genes as the blueprints for your body. When some parts are missing or there are mistakes in these blueprints, gene therapy may be used to correct those mistakes. Gene therapy is a process that involves…

Types of Cardiomyopathy

Five Main Types of Cardiomyopathy Dilated Cardiomyopathy (DCM) Dilated Cardiomyopathy (DCM) is found most frequently in adults under 50 years old. DCM causes the heart muscle to stretch and become thin, which makes it difficult for the heart to pump blood, leading to heart failure. In close to half of all DCM cases, the cause is unknown and most likely has a genetic basis or is inherited. In some cases, DCM can also cause problems with the heart’s electrical system which may interfere with the heart’s performance and can cause dangerous arrhythmias. To learn more about DCM, visit our partners…

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