Facing the Uncertainty That Comes With Having a Rare Disease

Life with LEMS brings unexpected challenges that require resilience

Lori Dunham avatar

by Lori Dunham |

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I think most people struggle to thrive in uncertain times. I know that in my case, uncertainty makes me anxious and often impatient. When faced with uncertainty, what normally would be a small annoyance becomes larger than life itself.

The Lambert-Eaton myasthenic syndrome (LEMS) community has had its share of uncertainty over the past few years. Every LEMS patient has a unique story of being diagnosed and living with a rare disease. Many had to fight for their diagnosis and then resolve problems with insurance companies, pharmaceutical companies, doctors’ offices, and sometimes even the justice system.

A number of people in our community have been affected in some way by the uncertainty created by a lawsuit between Catalyst Pharmaceuticals and the U.S. Food and Drug Administration (FDA) regarding the treatments Firdapse and Ruzurgi (both with the active ingredient amifampridine).

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Life’s curveballs

Our daughter Grace, who was diagnosed with LEMS at 14 years old, made the switch from Ruzurgi to Firdapse with only minor issues despite the anxiety the legal situation caused our family. I know that others didn’t fare as well as we did.

For a few months after that upheaval, we managed to settle into a familiar rhythm in regards to Grace’s health. She was doing very well with her medication cocktail of Firdapse, Mestinon (pyridostigmine), and Rituxan (rituximab) infusions.

Then, out of nowhere, came yet another fire to put out. It seems our health insurance has stopped paying for her Rituxan infusions. Grace has received these infusions every six months for the past two years. This treatment, in conjunction with amifampridine, has increased her quality of life in many meaningful ways.

Grace has gotten stronger and more balanced. With the addition of Rituxan infusions, she’s stopped gagging on her food and slurring her words. Her energy is back to normal, she sleeps better, and she has gained back the weight she lost during diagnosis.

However, uncertainty swirls again as we receive appeal letters and notifications regarding Rituxan payments.

I know uncertainty is a way of life for us because of Grace’s LEMS diagnosis. I’m aware that as she nears her 18th birthday, we’ll continue to face challenges thrown at us as we maneuver insurance and care into adulthood.

Additionally, treatments sometimes aren’t FDA-approved for a particular disease, so we must prove their effectiveness and need to insurance companies. Getting treatments off label also can sometimes prove challenging. All of this adds to the uncertainty of continuity of care. I wish it wasn’t this way, but it’s our reality.

I frequently have to talk myself off the ledge, so to speak. Last week, I found myself reciting the following as I confronted the insurance issues:

Have patience. Dealing with bureaucracy and red tape can be stressful. It may not get fixed today, but at least you made progress.

Take one day at a time. Do what you need to do today. Don’t worry about tomorrow.

Stop obsessing. Make the calls you need to. Talk to whomever you need. Email and fax whatever documents you can. Then stop.

Take a break. Refocus and enjoy the changing seasons. Read a book. Have coffee with a friend. Bake some cookies. Whatever brings you joy, add some of that to your day.

Be intentional. Make a list of what you need to get done that day in regards to health issues, then find something else to focus on momentarily.

Be kind. Even in the most stressful situations, I find that when we use our words in kind and gentle ways, we get quicker and more favorable results.

My nature is to assume that all of this uncertainty is a glitch and eventually will work out. If not, we’ll deal with it and come up with plan B.

“Worrying is carrying tomorrow’s load with today’s strength — carrying two days at once. It is moving into tomorrow ahead of time. Worrying doesn’t empty tomorrow of sorrow, it empties today of its strength.” — Corrie ten Boom 

Note: Lambert-Eaton News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Lambert-Eaton News or its parent company, BioNews, and are intended to spark discussion about issues pertaining to Lambert-Eaton myasthenia syndrome.


Anngail Norris avatar

Anngail Norris

I received an update yesterday from my medication supplier and was really shocked to see that the real cost of Firdapse is well over $50,000 a month. I have Medicare, supplemental insurance, and a grant to pay the balance. And I know that when I have received Rituxan infusions, it was almost $20,000 each time, which was paid for by Medicare and supplemental insurance. But what do people without Medicare or insurance do? And what happens if your insurance company won’t pay for your treatment? Who can possibly afford these prices? I know that AARP has been lobbying for lower drug prices, but if I had to even pay the $2,500 monthly co-pay, I would be out of luck. What do people who do not have Medicare do? In this case, I’m glad that I was old when I was diagnosed. But if you’re 18 and will need treatment the rest of your life, what happens? Keeps me up at night!


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