「Natural History Studies: Danon Disease」を編集中

<br>RP-A501 is being developed for Danon Disease, a rare genetic disorder characterized by thickening and weakening of the heart muscle, often resulting in heart failure, and for male patients, frequent death during adolescence or early adulthood. Rocket is the first company demonstrating safety and efficacy data in clinical studies for gene therapy targeting the heart. An open-label, global Phase 2 pivotal clinical trial of RP-A501 for the treatment of Danon Disease has commenced. The trial is expected to enroll 12 male patients from the U.S. EU. The trial will assess a co-primary endpoint consisting of LAMP2 protein expression and left ventricular (LV) mass reduction from baseline at 12 months. Natural History studies are observational studies that gather comprehensive data on the course of the disease to gain a better understanding of its progression and impact on patients. Data captured may include information on the physical signs and  [https://harry.main.jp/mediawiki/index.php/%E5%88%A9%E7%94%A8%E8%80%85:LakeishaDecicco harry.main.jp] symptoms patients experience, test results (for example blood tests or imaging), genetic information, and health-related quality of life, over a period of time.<br><br><br><br>This information helps describe the disease course and identify important factors (e.g. demographic, genetic, and/or lab test results), that may contribute to better or worse disease outcomes. Travel, accommodation, and food stipend would be provided. Study assessments are provided free of charge. Natural History Studies are particularly important for rare diseases. Given the diversity of symptoms and the small size of the patient population, these studies are often the only opportunity for researchers and physicians to address significant knowledge gaps and obtain a robust understanding of the disease and patient experience. Understanding characteristics of the patient population to define a treatment benefit from a patient’s perspective. Provide patient-centric data for health authorities such as the FDA, and other public health institutions to use in their review processes of investigational treatments. Interested in learning more? To read our Expanded Access statement, click here. RP-A501 is being developed for Danon Disease, a rare genetic disorder characterized by thickening and weakening of the heart muscle, often resulting in heart failure, and for male patients, frequent death during adolescence or early adulthood.<br><br><br><br>Rocket is the first company demonstrating safety and efficacy data in clinical studies for gene therapy targeting the heart. An open-label, global Phase 2 pivotal clinical trial of RP-A501 for the treatment of Danon Disease has commenced. The trial is expected to enroll 12 male patients from the U.S. EU. The trial will assess a co-primary endpoint consisting of LAMP2 protein expression and left ventricular (LV) mass reduction from baseline at 12 months. Rocket is sponsoring a multi-center, global, non-interventional natural history study designed to collect longitudinal prospective and retrospective clinical information on male and female patients with Danon Disease. The overarching goal of the study is to characterize the natural history of Danon Disease by collecting de-identified information from patients living with the condition. Initial trial sites for the natural history study in the U.S. EU have been identified and are currently enrolling patients. If you are diagnosed with Danon Disease or a family member and are interested in potentially enrolling in the Danon Disease Natural History Study, please contact us.<br><br><br><br>Natural History studies are observational studies that gather comprehensive data on the course of the disease to gain a better understanding of its progression and impact on patients. Data captured may include information on the physical signs and symptoms patients experience, test results (for example blood tests or imaging), genetic information, and health-related quality of life, over a period of time. This information helps describe the disease course and  [https://www.ncue.net/bbs/board.php?bo_table=free&wr_id=143662 site] identify important factors (e.g. demographic, genetic, and/or lab test results), that may contribute to better or worse disease outcomes. Travel, accommodation, and food stipend would be provided. Study assessments are provided free of charge. Natural History Studies are particularly important for rare diseases. Given the diversity of symptoms and the small size of the patient population, these studies are often the only opportunity for researchers and physicians to address significant knowledge gaps and obtain a robust understanding of the disease and patient experience. Understanding characteristics of the patient population to define a treatment benefit from a patient’s perspective. Provide patient-centric data for health authorities such as the FDA, and other public health institutions to use in their review processes of investigational treatments. Interested in learning more? To read our Expanded Access statement, click here. 2025 Rocket Pharmaceuticals. © Rocket Pharmaceuticals, Ltd.<br>