Baby receives new gene therapy for spinal muscular atrophy

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CHATTANOOGA, Tenn. (WDEF) — A baby at Children’s Hospital at Erlanger recently received a brand new gene therapy for spinal muscular atrophy.

Willie and Ashton Clayton look at their daughter Delanie after a doctor’s appointment.

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Her mother says after the six month old was born they noticed she lost weight, had no head control and no movements in her wrists.

They learned she had spinal muscular atrophy.

“The day she was diagnosed was definitely the worst day of our lives and then to come around and say, but there is hope,” Ashton Clayton said.

Delanie was the first baby at Children’s Hospital at Erlanger to try a brand new gene therapy worth $2.1 million.

“Historically it has been a really devastating diagnosis for children and their families. It has a progressive neurodegenerative course where the children just get weaker and weaker. The early onset disease typically would culminate death usually by two years of age. This brand new intervention provides hope for these families and seems to be helping retain strength beyond what would otherwise be expected,” Dr. Lewis Miller said.

Doctors are pleased with how it has gone so far.

“I have been pleasantly surprised with the small numbers that we have had, with some gains in just some minor strength,” Dr. Miller said.

Delanie parents say this gives her a new lease at life.

“so as nervous as we were, we talked about with our family all of the time, this is just as big as her birthday. This is something that we will celebrate every year for the rest of her life that she got a second chance,” Ashton Clayton said.

For other parents going through this with their child, they say there is now hope.

“I mean you have to not take no for an answer and you have to fight everyday to get the care that they need,” Ashton Clayton said.

Doctors say for a month Delanie will continue a steroid course to prevent adverse reactions from the medication.