LAMP2 reStart
Gene Therapy Study
Check if you are ready to participate:
If your family member has been diagnosed with Danon Disease through genetic testing, the results would have revealed a missing or damaged LAMP2 gene.
In this study, an experimental one-time treatment delivers a working copy of the LAMP2 gene into the body's cells. Once inside the cells, particularly heart muscle cells, the working gene can produce the normal LAMP2 protein, which is needed to clear the cell’s waste and restore normal processes.
Because the IV delivering the correct LAMP2 gene needs to come through a modified virus (able to penetrate the cells quickly and has been made harmless) participants are often given immunosuppressants for a while before the treatment, so the body’s immune system doesn’t attack the harmless virus delivering the needed gene.
How to Participate
The first step is to be interviewed by the Support team. They will ask about the diagnosis, symptoms, current medications and procedures. They may also ask you if to get the medical records for the study site’s medical team.
If there is a match to the requirements of the study, the Support team will ask about which study site you’d like to go to, and then make contact with the site to refer you.
The second step is to be interviewed by the team at the study site, who will review the medical history with you.
The next step is for testing at the study site, which could take a few days. During the time at the study site, you will be provided with support and your expenses will be paid for by the sponsor.
The investigational gene therapy will be given in one dose, by IV infusion. After that, you can return home, but the participant will continue to be monitored for a period of time by the study medical team.
Understanding the levels of Heart Status
Do you know your heart status?
The NYHA system ranks heart failure in four levels, based on how much symptoms limit a person during everyday physical activity.
Knowing Heart Function Status
Check which level best applies to you or the person you care for:
Class I: No limitation; ordinary physical activity does not cause much fatigue, shortness of breath, or heart palpitations.
Class II: The person can do normal activities, but they get tired, short of breath, or feel their heart race more easily than usual.
Class III: It’s hard to do normal activities because you get symptoms like tiredness or shortness of breath very quickly.
Class IV: You feel tired, short of breath, or uncomfortable even when resting. Any activity makes you feel worse.
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