Danon Disease?
Research can open a new path of understanding of how to slow disease progression
New research in Danon Disease seeking participants
Danon Disease is a rare genetic disease which happens because of a change in or a missing LAMP2 gene, which makes it harder for cells to clean up waste. The buildup of waste in the cells can affect multiple body systems, but because is can increase thickness in the muscles of the heart it can become life-threatening.
Currently, the aim of treatment is to prevent sudden cardiac death and reduce fatigue and other symptoms. Other than a heart transplant, there is no approved treatment available to slow the progression of this devastating disease.
Joining a research study provides the chance to help in the development of an effective treatment and to come under the care of the leading Danon disease specialists for the duration of the study.
Who can participate in the Natural History Study?
Biological Males, Age 8 years or older
Biological Females, Age 8 and older, and under age 35
Those with a diagnosis of Danon Disease, confirmed by a genetic test
Those who are willing to provide access to medical records / medical history
DANON hiSTORY A Natural History Study
Because Danon Disease is such a rare condition, there is little information for researchers to study to help them understand the disease and how it may be treated. Researchers need to learn from the real-life experience of people living with Danon Disease, and by joining the LAMP 2 hiStory Study, you can provide the information needed.
The goal of this study is to collect data of the signs and symptoms, key events, and the impact of the disease on quality of life, as it is managed with current treatments.
Participation does not add any new therapy, only access to your medical history and collecting your feedback through surveys and interviews with the study team.
DANON VIVO
Clinical Study for an Investigational Gene Therapy
Gene therapy is being developed for Danon disease, with the investigational therapy designed to deliver a functional LAMP2 gene into a patient's cells to replace the damaged gene.
The goal of this clinical trial is to assess its long-term safety and effectiveness in improving heart function and other symptoms.
How is the gene therapy supposed to work?
Replacing the gene: Danon disease is caused by a mutation in the LAMP2 gene, which prevents cells from properly recycling waste. The investigationalgene therapy delivers a working copy of the LAMP2 gene into heart cells, so the cells will be able to clear waste properly.
Single-dose infusion: Treatment is given once, as a single intravenous (IV) infusion.
To download a fact sheet about the reStart Gene Therapy Clinical Study click here.
Who can participate in the Gene Therapy Study?
Biological Males, Age 8 years or older
Those with a diagnosis of Danon Disease, confirmed
by a genetic testNY Heart Association
Class II or Class III
Frequently Asked Questions
What is needed for a Danon’s Disease diagnosis? A genetic test, ordered by a geneticist or another specialist, can detect whether there is a damaged or missing LAMP 2 gene.
What is the difference between a natural history study and a clinical study? Collecting data about a rare disease like Danon Disease is necessary to understand how it starts, its cause, and how it progresses, in order to be able to develop a treatment for it
How do we find out if we can participate?
Fill out and submit the form and if you match the study requirements you will be contacted by the support team who will help you connect with the study site team.
If we join the Natural History study, can we join the gene therapy study at a later time?
Yes, if the person you care for starts off in the natural history study, and is eligible and willing to participate in the gene therapy study, you can apply for a prescreening interview with the Re:Start Study team who will decide if you are eligible to participate in the gene therapy study.
Are there any costs to participate in either study?
The costs of the therapy and visits will be covered by the study sponsor, as is travel to the study sites.
What happens if we change our mind about participating?
Participation is completely up to you and your family. You can withdraw permission to access records from the Natural History study, and discontinue the process of screening and testing for the gene therapy without any problem to you.
Ready to Participate?
To contact us about help in participating in either study, leave your contact information, and a message, and our Support team member will follow up with you shortly.
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