Health Beat

Health Beat: New hope for spinal muscular atrophy

ORLANDO, Fla. - Asher Camp is now four-and-a-half years old. He was diagnosed with spinal muscular atrophy at just six months.

"For days, we were crying, because we just knew what we were about to face," said Amanda Camp, Asher's mom.

"What we've learned through the clinical trials with Spinraza is that definitely the drug improves both survival and the ability to improve in their motor function," said Dr. Richard Finkel, the chief of neurology at Nemours Children's Hospital.

Patients with SMA have a flaw in their SMN1 gene, so they rely on their SMN2 gene, which does not produce enough protein for proper muscle function.

"So what Spinraza does, is it works on this SMN2 gene to try to increase the amount of protein that's made and try to restore function," Finkel said.

Spinraza, or Nusinersen, was approved by the FDA in December 2016. The results have been transformative for Asher.

"He is doing things like crawling and sitting up. He's able to balance himself while standing," said Camp.

Finkel said early diagnosis and treatment are key.

"Hopefully, the word is getting out to neurologists who would be caring for these babies to say that SMA is now a treatable disease," Finkel stated.

There seem to be no negative side-effects to the drug, however, long term trials are still being conducted.

Spinraza costs about $125,000 per dose. It is covered by most insurances and state Medicaids, and must be administered by spinal tap every four months in a hospital or clinical setting.


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