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About
Danon Disease
Danon Disease is a rare X-linked condition caused by a missing or mutated LAMP2 gene, leading to waste buildup in heart and muscle cells. It commonly causes thickened heart muscle, muscle weakness, and reduced cognitive abilities—symptoms that are often more severe in males.
Currently, the aim of treatment is to prevent sudden cardiac death and reduce fatigue and other symptoms. During the past ten years, research has helped to increase the understanding of Danon Disease to enable the development of potential treatments to slow or reverse the progression of the damage to the body’s organs.
At Know Rare, we help people living with rare conditions like Danon Disease find clinical trials, support, and resources that fit their needs.
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Frequently Asked Questions
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A clinical trial is a research study designed to evaluate the safety and efficacy of new medical treatments, interventions, or therapies. These trials aim to advance medical knowledge, improve existing treatments, or develop new ones. Participants in clinical trials play a vital role in advancing medical science, and their involvement is based on informed consent and ethical guidelines.
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Clinical trial participation provides access to cutting-edge treatments, potentially improving health outcomes, while contributing to medical advancements. Close monitoring, and personalised care from leading experts are other potential benefits.
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Clinical research is voluntary. Individuals have control over their decision to participate and are able to withdraw at any time. However, participants should recognize the responsibilities involved and consider whether they can meet the requirements before joining a study. Study centres aim to provide confidence through thorough pre-screening and screening, ensuring questions are answered, and expectations are clear.
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Our patient support team will call you to review your health history or the health history of your loved one to assess suitability for a clinical study. If there aren’t any current matches, we'll keep you informed about potential opportunities for the future.
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Think of Know Rare as your personal navigators to clinical trial information and leading disease experts. With a team experienced in medical and clinical research, we can help you understand eligibility for potential clinical trials and connect you with nearby research centers. Our understanding of the clinical trial process ensures you have a clearer idea of what to expect throughout your journey.
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There is no charge to use our services. Our mission is to create awareness about ongoing clinical trial opportunities and make them more accessible for people living with rare diseases. Our company founders, and many within our company, have walked a path of uncertainty in the face of a rare disease diagnosis, and we genuinely care about others and strive to do what we can to simplify the clinical trial process.
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One of the services Know Rare provides is to help you explore participation in clinical studies. We created this service to help make joining a study an easier, more productive experience. Know Rare has access to rare disease clinical studies listed on the FDA’s website clinicaltrials.gov. Based on your profile, which you can fill out with the help of our team, you can be directed to a clinical study that may be right for you. The team can help you make contact with the researchers at a study centres of your choice. Your personal health information and contact information will not be forwarded to anyone else, including the study sites, without your specific permission.
The goal of Know Rare is to help make it easier to connect people to clinical studies. Study sponsors help in this effort by providing the funding for our efforts and by helping us get in contact with researchers and the study sites. Though the sponsors provide funding to help us connect people to their clinical studies, please know the choice of which study you wish to participate in is up to you and your doctors.
“Researchers are interested in what you have to say, so they can learn more about your rare disease.”
Lynn
Mother of Casey, diagnosed with a rare metabolic disease
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