A Colorado mom is on a mission to raise money for her daughter’s type 1 spinal muscular atrophy (SMA) treatment — that would save her child’s life.
What is Spinal Muscular Atrophy?
Affecting approximately 1 in 10,000 people, spinal muscular atrophy (SMA) is a rare genetic disorder that causes mild to severe muscle weakness and degeneration. The types of spinal muscular atrophy vary based on severity and age the condition was developed.
Type I (Werdnig-Hoffman disease) — which is the type Maisie has — is a severe type diagnosed at or soon after birth. It causes developmental delay and inability to sit or support the head independently. It also causes breathing and swallowing issues.
Treatment vs Treatment
This is the reality for Maisie and her family:
“She can’t be left alone ever, because in a second she can choke on her own secretion. It just takes one cold to kill them. We lost Maisie twice. We’ve had to resuscitate her two times so far in her life,” said Ciji, Maisie’s mom.
Ciji regularly has to conduct what’s called a cough assist—as well as a “suction,” that gets rid of excessive secretion from Maisie’s mouth.
“It’s pretty horrible, gruesome stuff,” she said. “As a parent, it’s nothing that a parent should have to ever do to their kids.”
That is why Ciji is doing everything she can to raise the $2.2 million needed for the FDA-approved treatment for children under 2 years old — a new gene therapy called Zolgensma.
This treatment not only halts disease progression, but can ultimately reverse symptoms over time.
“This is my baby and I want to give her the best chance at life, and I can’t do that,” she said. “I can’t do that because I don’t have $2.2 million.”
According to Ciji, Medicaid denied Maisie for Zolgensma because she is already using another drug, Spinraza (another treatment that Ciji says just slows the progression of SMA, not stops it, like Zolgensma.).
“She used to not be able to move her arms as well,” said Ciji. “But her breathing continues to decline and that’s why we want the gene therapy because it halts the progression [of the disease].”
Currently, she said Medicaid covers the cost of a Spinraza injection, given every four months at a cost of $125,000.
Ciji doesn’t waste a second underlining the urgency at hand; she says Maisie could be running out of time.
“In May, I watched five of my friends bury their babies — all on Spinraza, all around Maisie’s age.”
In an interview with her local news station Ciji pleads with her community to help her raise the money before it’s too late.
“Please,” she said with tears in her eyes. “This is my baby, and I would do anything for your baby. What would you do if it was yours?”
And when she puts it this way, it really makes sense.
“I could find $2 in the bottom of my purse or in my car. If we could get a million people to find those two dollars, we could do this.”