INDIANAPOLIS, Ind. - Family of 4-month-old Anthony Schmitz has more hope after the boy received expensive treatment that could improve his quality of life.
Anthony is fighting a terrible disorder called Spinal Muscular Atrophy, or SMA. It can kill kids before the age of 2. (SMA). It is the leading genetic cause of infant mortality.
Children with this condition have problems holding their heads up, swallowing and breathing. These symptoms may be present at birth or may present by the age of 6 months.
The medicine that could help him live longer costs more than $2 million. In 2019, the U.S. Food and Drug Administration approved Zolgensma, the first gene therapy approved to treat children less than two years of age with spinal muscular atrophy.
Anthony's mom, Louise Johnson, said after Indiana Medicaid denied her claim, she filed an appeal and the program approved it.
Her son received the medication on Wednesday.
"Amazing," she said. "I want to see him live forever. No mom wants to bury her child, so I just want to see him grow up with his brothers and play with his brothers."
Johnson has three other sons and none of them have SMA. Anthony has been hooked up to machines at Riley Hospital for most of his life. Family and doctors hope Zolgensma will help the boy live longer and improve his quality of life.
"Not knowing when we were going to say our goodbyes or how long we would have with him," Johnson said. "I am hoping one day he can breathe on his own without a ventilator. That is my hope for him."
Johnson only makes trips back home to Evansville every two weeks. Now, they just have to wait and see if this treatment will work.
"Does his respiratory function improve? Is he able to work more fully without as much ventilator support?" Dr. Larry Walsh said.
Dr. Walsh said this expensive medication was Anthony's best option. He said he is optimistic.
"When he does not need to be in a ventilator, that would be a tremendous win for his family," he said.
Family is nervous about the unknown. Yet, his mom hopes her son's future includes leaving the hospital soon with a second chance at life.
"It a chance at life," Johnson said. "With a better quality of life."
Like in this case, many medications require prior-authorization from Medicaid before they can be used. Indiana Medicaid said criteria used for prior-authorization is based on clinical evidence only and cost is never a factor. The program said it has authorized the use of this specific medication on four occasions.