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How much would you pay for a miracle?

Yesterday's New York Times featured an editorial about the cost of gene therapy and the recent controversy surrounding Norvatis's gene therapy for SMA.

Here is co-founder, Maria Kefalas' response to the New York Times.

Today’s opinion piece on the gene therapy pricing debate was disappointing, to say the least. You offer a definition of SMA that looks like it was lifted from Web MD and then you launch into this same old pricing debate and diatribe about the FDA. You also did not speak to one single patient family. So, please let me address the gaping holes in the piece.

My youngest daughter Cal has metachromatic leukodystrophy or MLD. MLD is in the same family as Tay-Sachs disease, if ALS and Alzheimer’s disease had an offspring, that could be MLD.

My daughter Cal is dying. For the last 7 years I watched my chubby toddler who loved meatballs and soccer and Kipper the dog lose the ability to walk, talk, feed herself, use the potty and see.

We could not celebrate her 3rd birthday with a cake because she had just gotten a feeding tube. It felt cruel to have cake she could no longer enjoy.

The day that she was supposed to start kindergarten, I lay in bed all day crying.

In 2017, I spent Christmas and New Year’s Day and my daughter’s birthday in the PICU hoping that Cal would manage to be able to breathe without a ventilator again. That hospitalization cost the taxpayers more than $100,000 because she receives Medicaid to supplement the costs of her care Blue Cross won’t cover.

In 2018, I could not drop off my oldest child to college because my husband and myself cannot leave our daughter Cal overnight because we lack nursing coverage. I went to the beach once in two years. We celebrated last Thanksgiving eating macaroni and cheese because Cal got sick again and had to be readmitted to the hospital.

My husband and I have not been away on a vacation together since our Make A Wish trip in 2012. I don’t go to my son’s basketball games and missed my oldest daughter’s cross country meets even when she was a four-year varsity runner and captain of her team. I will confess to not knowing the names of my older children’s teachers because I am too busy running a hospital with a full-time staff of nurses out of my home.

The parents of terminally ill children experience more depression, divorce, and lose wages and promotions. One paper I read suggested that parents of sick and disabled children die younger than other parents. Such things cost society money, wouldn’t you say? Many researchers have documented the trauma to young children who must watch a sibling die.

But, the kids with my daughter’s disease treated with gene therapy are going to school, playing soccer, singing, dancing, eating pizza and enjoying remarkably normal lives. Those families don’t spend Christmas in the PICU. Instead, they get to go to the movies and take a vacation and drop off their kids to college and eat birthday cake to celebrate birthdays. Most importantly, they don’t have to plan their little girl’s funeral.

How much is that worth?

When doctors performed the first heart transplant or did the first bone marrow transplant, I don’t believe the New York Times wrote an editorial piece complaining about the price tag for a genuine miracle.

Shame on you.

Maria Kefalas, The Calliope Joy Foundation and Cure MLD

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