Consigue ayuda para encontrar las últimas investigaciones sobre la enfermedad de Danon
Póngase en contacto con nuestro equipo de asistencia en directo para ponerse en contacto con los principales especialistas, acceder a estudios de investigación y participar en ensayos clínicos sobre la enfermedad de Danon
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Nuestro equipo se dedica a orientar a las familias hacia investigaciones fundamentales, especialistas y ensayos clínicos. Ofrecemos orientación, consuelo y apoyo.
Know Rare's global impact is a testament to its team who have been impacted by rare diseases and are focused on turning their pain into purpose, touching countless lives with empathy and determination.
In need of care and support?
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La enfermedad de Danon
La enfermedad de Danon es una afección poco frecuente ligada al cromosoma X, causada por la ausencia o mutación del gen LAMP2, lo que provoca la acumulación de residuos en las células cardíacas y musculares. Suele provocar un engrosamiento del músculo cardíaco, debilidad muscular y una disminución de las capacidades cognitivas, síntomas que suelen ser más graves en los hombres.
En la actualidad, el objetivo del tratamiento es prevenir la muerte súbita cardíaca y reducir la fatiga y otros síntomas. Durante los últimos diez años, la investigación ha contribuido a mejorar el conocimiento de la enfermedad de Danon, lo que ha permitido desarrollar posibles tratamientos para frenar o revertir la progresión del daño en los órganos del cuerpo.
En Know Rare, ayudamos a las personas que padecen enfermedades raras, como la enfermedad de Danon, a encontrar ensayos clínicos, apoyo y recursos que se adapten a sus necesidades.
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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 personalized 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 centers 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 center 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.
«A los investigadores les interesa lo que tienes que decir, para poder aprender más sobre tu enfermedad rara».
Lynn
Madre de Casey, a quien le han diagnosticado una enfermedad metabólica rara
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