A 16-month-old baby boy was denied a possible cure of what is touted as one of the most expensive drugs in the world. Little Axel Dennis was diagnosed with Spinal Muscular Atrophy (SMA) Type 1 when he was only 5 weeks old. SMA is a genetic disorder that afflicts an individual’s muscle growth and movement.
Axel’s mother, Andrea James, said that her son’s muscles were not receiving messages and signals from the brain, and hence were unable to carry out their locomotive abilities naturally.
We were told one to two years and bring him home and enjoy him the best we could, but there were no guarantees of his life span, Andrea said.
Earlier this January, Axel slowly lost the ability to smile as his face muscles just couldn’t do it anymore. It was around the same time when the FDA approved the drug Zolgensma for children under the age of two who were suffering from SMA.
The infusion stops the disorder from developing in its tracks and inhibits the progress of the detrimental condition. Zolgensma is a single-dose drug that has been praised by the SMA community, but the major drawback is that it’s insanely expensive, costing over $2 million. Unfortunately for Axel, his Louisana Medicaid had refused to cover the cost.
We’ve been denied twice and now we’re applying for the State Fair Hearing. I think that they just don’t want to pay the price. The money’s been spent to prove that it’ll benefit these kids, she said.
It will save their lives, as close to a cure as we’ve come and as what anything could be really. I say to the state, save his life, give him a chance to walk, give him a chance to earn his smile back.
In the letter the family received which denied Axel’s treatment, it was stated that the 16-month-old boy didn’t meet certain necessary criteria in order to receive the dose. His mother Andrea said that it was actually because he has a trach tube, which is used to help expand Axel’s lungs.
Now the family is racing against time itself. They have only eight months before Axel turns two, after which the FDA would not approve the drug for him, or anyone else. Andrea said that she has heard and seen what the drug can do, and truly wishes that everyone suffering from SMA is treated with it.
The Louisiana Department of Health stated that their hearts go out to parents with children suffering from a life-threatening disease such as SMA. Louisiana Medicaid has assured that they will indeed cover all treatments for SMA that have been approved by the DFA and also by review cases to ensure that the child meets the necessary medical requirements.
As Axel fights for his life, his treatment continues with Spinraza, which had been approved by the DFA back in 2016. He is administered the drug every four months through spinal injection and must undergo anesthesia to receive the treatment.
As Axel deals with this terrible ordeal, Andrea and the rest of the family have set up a GoFundMe page and a Facebook group to help raise money and awareness for his condition.