Living with
Danon Disease

Questions for your physician

Any new diagnosis comes with a lot of questions, especially after you get your initial diagnosis. Through conversations with patients and caregivers, we summarized our list of questions that might be helpful to discuss with your physician, geneticist and/or cardiologist. Please note that this is not comprehensive, but may be useful as a starting point.

  • What does my diagnosis mean for me and my health?
  • How does this affect my family members? Should I get any of them tested for Danon Disease?
  • What other tests do I need? What other specialists should I go see (and when)? Could you refer me to a trusted colleague that specializes in…?
  • What type of care coordination services does your office (group, hospital, health system) provide?
  • How often do I need to have a follow-up appointment?
  • What do I need to look or watch out for? What information do I need to track and how often between visits to help with better management of my care?
  • What does this mean for my kids?
  • What implications does this have if I want to have more kids?

Managing school with Danon

Having a child with Danon Disease can be challenging . It’s difficult to know how Danon Disease will affect each child. Managing the disease can already be scary and frustrating — and then you throw school into the mix. We know first hand that navigating the school system with a child with Danon Disease can be overwhelming. Who can you talk to? Where do you start? What accommodations do they need? What classes do they need? What therapies do they qualify for in school? How do you explain this to their educators?

First, provide your child’s school counselor with any information you can on Danon Disease, including a physician’s letter with your child’s diagnosis. They may have a lot of questions to better understand how to help your child — try your best to answer them and, if you don’t have the answer, take notes so you can follow-up afterwards. While you should discuss any questions with your child’s care team, you can also reach out to us — we may be able to help.

Then you will need to request an Individualized Education Program (IEP) or a 504 Plan for your child. More information can be found here to help you determine which plan is most suited for your child's needs. If you can, it is also helpful to have your child receive neuro-psych testing at a children’s hospital or outside facility. Bring the results summary with you when approaching the school — these results inform the school on what specific supports and accommodations your child will need moving forward. If possible, it may be helpful to include information about any specialists your child has seen, including any visit notes.

Danon Disease is rare — educators and many health care providers will be unfamiliar with it. Teachers, staff, and school nurses may not be prepared when a child with a rare disease like Danon Disease comes into their school. Unfortunately, this puts the burden on you and your family to advocate and educate the school. One parent shares, “I always wrote a letter at the beginning of each school year to my child’s teachers, staff and school nurse. I explained the disease, how it affects him, where he was with his health and the possibility of him missing school due to appointments, illness, surgeries and/or just not feeling well. I gave them my contact information and always asked that they contact me if they saw any changes in his mood, behavior or daily routines.” One Danon Disease parent noted that the open communication with the teachers and staff really helped them understand and feel like part of the child’s team.

Advice for caregivers

As a caregiver, it can be physically and emotionally challenging to care for a person with Danon Disease. In some cases, caregivers do not have Danon Disease, and in others, they share the disease with those that they are caring for. Either way, it is hard to manage all of the appointments, surgeries, schedules, school, medications, therapies, etc. — the list goes on.

The level of care required can be quite costly. While some families may have access to some type of health insurance (private, Medicaid, etc.), state and public programs may provide additional financial or program services support, for example, disability benefits such as Supplemental Security Income (SSI), homes health services, help with the cost of copays and medications. If you aren’t sure where to start, sometimes contacting your local county or state can be a good start.

For those of you caregivers that are also patients, you’re not alone — some of us are in the same boat! This is a hard job. There will be days where you don’t feel good, but still you have to get up, cook dinner, make lunches, give baths, make sure meds are taken, appointments are made, and work a job. Danon Disease is a genetic condition that often runs in families. This can mean balancing your own self-care and the care of your loved ones.

Take one day at a time. When you feel overwhelmed, reach out for support. You are not alone and, whether you have Danon Disease yourself or not, caregivers need support too. It is normal to feel overwhelmed at times. Visit the Facebook Support Groups to find more information about Danon Disease and connect with others who may have similar experiences:

Danon Disease Support Group
Danon Disease Advocacy, Support & Education

Talking about Danon

The first step in educating others is to learn as much as you can yourself. Check out support groups and our site, engage in conversations with researchers, physicians, geneticists and/or your cardiologist. This is our starter list of things that can help you explain Danon Disease to others:

  • Danon Disease is a rare, genetic condition, and can affect the heart, muscles, vision and learning
  • People with Danon Disease see many specialists — usually the cardiologist is the primary physician
  • Danon Disease is a lifelong disease and there is no cure. Currently, only treatments are available to help with symptoms and underlying issues
  • Danon Disease presents itself differently, person-by-person. Some have symptoms daily, others may experience them once a week, and some people may not have symptoms at all. Danon Disease can also progress quickly or over time