Boy with genetic disease gets $2.1M medicine after Medicaid’s initial denial

Boy with genetic disease gets $2.1M medicine after Medicaid’s initial denial

INDIANAPOLIS (WISH) — The future looks better already for a mother who spent months fighting to get a $2 million drug for her son with a genetic disease.

As News 8 reported Friday, she credits our interview with her for making it happen.

Anthony Schmitz got that infusion Wednesday morning.

It’s a day his mother Louise Johnson admits she didn’t think would happen.

But now, she likely must wait weeks before learning just how much this will help. That’s the real battle.

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Yet her battle to get the $2.1 million infusion is over. Even doctors are pretty happy to be a part of it.

Anthony is just 4 months old He has SMA-1, a severe form of spinal muscular atrophy in which motor neuron cells die without treatment.

The treatment is “a chance at a life with a better quality of life,” Johnson said.

His case is so severe one doctor told Johnson and her fiance it would be fine to let Anthony die. But, they would have none of that.

One of two treatment options is Zolgensma, genetic therapy just approved last year by the U.S. Food and Drug Administration.

“We’re hoping for a miracle,” she said.

Zolgensma uses a virus to work to replace the missing genetic material inside Anthony.

Indiana Medicaid denied the treatment initially because he’s too weak to be off a ventilator.

Two days after we talked to Johnson and weeks after she’d appealed the decision, she found out he was approved.

“I think when News Channel 8 called them last week and told them they were going to do a story, I think that reversed it,” she said last week.

Dr. Larry Walsh watched as the infusion happened. He is Anthony’s pediatric neurologist.

“It’s pretty anti-climatic,” he admitted.

It just takes one hour, but he believes this will change Anthony’s trajectory for a lifetime.

“This is a pretty wonderful thing to be a part of,” the doctor said.

With such a new drug and even less data involving patients with cases as severe as Anthony’s, initially physicians weren’t even sure the treatment would work.

But by doing their research, reaching out to experts, working with Indiana Medicaid and doing their own homework, the doctors decided it was worth a try.

“We are going into this with hope and optimism,” Walsh said. “But, we’re going to have to see.”

The $2.1 million price is enough to make anyone blink but not stop.

“A group decision said medically this makes sense for this child so the cost fell by the wayside,” the doctor said.

Baby Anthony’s mother said, “I’m excited about his future.”

It’s a future made possible through her tireless efforts, spending 28 days a month at Riley Hospital for Children, which is almost a 4-hour drive from her family in Evansville.

Her advice to others is to not give up.

“It’s not a death sentence, so just keep fighting. It’s a baby, keep fighting,” Johnson said.

Anthony is scheduled to have a tracheostomy next week to be a more permanent solution than the ventilator he’s been on since birth.

Johnson hopes to take him home for the first time in March or April and eventually remove the trach, too.

“I’m hoping he can one day breathe on his own without a trach or ventilator,” she said. “I want to see him live forever. No mom wants to bury their child. I just want to see him grow up with his brothers.”

Every child responds differently to the new drug, and Anthony is one of the most serious cases to get the infusion. Walsh hopes in another six weeks or so, doctors will have a better idea how Anthony will respond by measuring his limb movements and breathing abilities.

SMA is the most common genetic cause of death in infants with a diagnosis of one case in every 11,000 births.

Indiana Medicaid tells News 8 the cost of treatment is not a factor in authorizing it. This is the fourth they’ve approved Zolgensma.

Walsh said it’s only the fourth or fifth dose ever given at Riley, which also taken private insurance.