GRAND JUNCTION — A miracle coming through for a Colorado baby! Maisie was born with a rare genetic disease that left her family trying to find out how to cover the $2.2 million cost for a live-saving treatment. Just days ago, the insurance came through for the family.
Maisie was diagnosed with Type 1 SMA at 6-months-old. It's a disease her mother Ciji had not heard of at the time, but when she got the news she knew it was not good.
"The Doctor put her hand on my hand, and I lost it, I knew in that moment that it really something bad and I was like 'why are you touching me? Dont’ touch me.'" Ciji goes on to say that at the time the doctors did not know of any treatments for the terminal disease.
Spinal muscular atrophy is a very rare genetic disorder. The muscles needed for speaking, walking, breathing and swallowing are destroyed. The brain stays intact, but eventually Maisie will lose the ability to eat or even breathe. Most babies with SMA don't live past the age of two. Maisie is 19-months-old.
Maisie has been getting a drug called Spinraza to slow the progression. This requires a spinal tap, without pain meds. The good news is the Food and Drug Administration recently approved a gene replacement therapy called Zolgensma. It has proven to halt the progression and even reverse symptoms. It's only available to children under the age of two.
This is their only shot to save Maisie's life and they been through a number of hurdles already. Maisie was in the hospital for 9 months when Zolgensma came about. She was diagnosed 7 days past the trial date. It was then extended, but the weight limit was 8.7 kg. Maisie was 9 kg. The trial opened up to kids under one. Maisie was four days from her first birthday.
Before hearing the shocking turn around from the insurance company approving the claim to cover the hefty price tag, the family was asking for big donations in a short period of time.
"I am begging, I’m literally out of options, we can sit there and be mad at the drug companies, make you be mad at the insurance company it that doesn't change the fact that right now she can't get it without the money," Ciji pleaded.
Maisie is just one story for one child with a miraculous ending. Not every child gets that opportunity. Now, her family plans to create a non-profit to help others along a similar journey with the goal of adding testing for SMA on newborn screenings.
To learn more about the effort, visit the GoFundMe first setup to raise funds for the medication.