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To Our Future President, Please Consider the Needs of Rett Syndrome Families

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Dear President-elect Trump,

I know you will soon be busy thinking about the economy, world conflicts, and trying to deal with all challenges your future position will ensue. But, if you could take a moment to focus on something else for a second, I would like you to make an effort to listen to those who are part of the rare disease community.

According to statistics, there are 25 to 30 million people affected by a rare disease in the United States. This means an average of eight to 10 percent of the entire population. They need your attention, as life hasn’t been easy for them so far, and it doesn’t look like they will get better any time soon.

I want to tell you a story about a little girl called Susan, who is currently diagnosed with Rett syndrome. This condition is so rare that a doctor may have a fulfilling long career without ever meeting a girl suffering from RS. The latest statistics say the syndrome affects approximately one in every 10,000-15,000 live female births (as it is known to affect mostly women) and there are currently around 30,000 RS patients across the U.S.

The problems Susan, her family and all other people affected by RS are pretty much the same: it impairs the development of young girls, causing speech problems, cognitive deficiencies, autism-like behavior, motor skills impairments, sleep disorders, seizures, feeding issues, total loss of walking abilities later on, and partial or total incapacity of taking care of her own needs. And, just like it happens with other rare diseases, like the Jervell and Lange Nielsen syndrome, a parent of an RS-affected often child has to prepare themselves to witness their child’s premature death.

If you never had to care for a child with a rare developmental disease, it is probably difficult for you to understand how each day can turn into what feels like a battle for survival for families and the affected children. I want you, President-elect Trump, to consider this, as I am sure you can do something to change things for the better.

Increase awareness

Parents with RS-affected children often have to become spokespeople and educate teachers, other parents and other kids regarding their child’s health issues when she attends school. This became very difficult for Susan’s mother, who was already pushed to her max, as having a rare disease in the family often means caregivers and patients experience increased exhaustion, depression, and depletion of the family budget.The responsibility of educating teachers and professionals in the field shouldn’t fall solely on the parents.

National awareness programs that address doctors, nurses, teachers, parents and children regarding RS patients should play a bigger role. Judgment, prejudice, labeling, stereotypes, discrimination and refusal to offer proper care are not new things in our society, unfortunately.

Susan was often called a “monster” and refused proper attention when integrated into a regular school environment. Susan’s mom had to write an individual letter to all other parents and explain her daughter’s condition, her needs and beg for her daughter to be accepted by her peers and their parents. This is why awareness programs that advocate for families with medically complex children should be readily available for those who need them.


This has been a sensitive issue in the past years. Now, imagine how hard it is for parents to provide their children with adequate care when they face the disturbing truth: for some, it is hard, if not impossible, to receive proper coverage. Some may obtain it, but it partially covers the broad spectrum of needs. Some parents lose the coverage when they change jobs, and others had their policies dropped when the insurance company deemed the disease as “pre-existing.”

Susan’s health needs are not fully covered, which means an additional struggle for the family, along with balancing the daily care for a medically complex child and mind-grinding jobs to pay the bills.

Day habilitation programs

In theory, it sounds simple: Susan’s mom just needed to pay a visit to the state’s Developmental Disabilities Agency and find a day habilitation program in her area. Unfortunately, the closest one required commuting. In this case, Susan’s parents were recommended to start their own local program and get other families involved while trying to access local funding. Needless to say, such endeavor proved impossible for Susan’s parents and given how things are. It is certainly just as difficult for other parents as well.


Even if Susan is only 9 years old, her parents started planning for the future. They are not getting any younger, and their daughter has some chances of reaching adulthood. This is why they need to think about guardianship, establishing a “special needs trust,” paying for a lawyer to deal with the legal work, application for Supplemental Security Income as she turns 18, etc.

The problem is that even if they receive the SSI benefits, the payments will still be backdated, as it takes around six months (if not more) to get the paperwork approved. The biggest problem is that those six months can end up with the parent burying and mourning their child before it’s resolved. It is a harsh truth, but all such families live with the mind-numbing idea that their child may die before reaching adulthood.

Funding for research

To date, there is no cure available for RS children. Doctors usually prescribe medicine for some of the symptoms. I know that as president, you cannot personally get involved and finance further research on comprehensive Rett syndrome cures, but you could at least encourage such further findings and results. Looking at the current data, there is some promise of partial reversal of RS-like symptoms in mice, but science could only go so far. Statistics show that we don’t have any information on adult RS women past the age of 40 and we can’t make reliable predictions regarding these patients’ life expectancy.

It is easy to ignore Rett syndrome children and adults, as they are not many. But it is my strong belief something can be done for them. Some of these children aren’t able to personally address you, and many of their parents are too exhausted to even consider the possibility. Therefore, I consider it to be my duty to advocate for those who don’t have a voice.

They struggle, but they never lose hope, cherishing every single day of their life.

Maybe it’s time to give those hopes a solid, unified foundation.

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Originally published: November 11, 2016
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