{"id":240663,"date":"2021-02-28T09:38:42","date_gmt":"2021-02-28T15:38:42","guid":{"rendered":"https:\/\/www.liveaction.org\/news\/?p=240663"},"modified":"2021-02-26T15:20:02","modified_gmt":"2021-02-26T21:20:02","slug":"baby-spinal-muscular-atrophy","status":"publish","type":"post","link":"https:\/\/archive.liveaction.org\/news\/baby-spinal-muscular-atrophy\/","title":{"rendered":"Thanks to prenatal screening, newborn receives life-saving treatment for spinal muscular atrophy"},"content":{"rendered":"
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At just four days old, a baby in Baton Rouge, Louisiana, has become the youngest person to receive a cutting-edge life-saving treatment \u2014 one of the most expensive in the world.\u00a0<\/span><\/p>\n

Lexa Dennis was diagnosed with spinal muscular atrophy (SMA) in the womb. Her older brother Axel also has the genetic condition, and her parents knew there was a 25% chance Lexa would too. <\/span>SMA is a <\/span>degenerative disease<\/span><\/a> that happens when a specific gene that makes a protein for motor nerve cells is missing. Without the gene to make the protein, motor nerve cells start to die. When that happens, bodily functions like eating, breathing, and moving can become difficult or impossible. The earlier symptoms appear, the more harmful the condition is. Pediatric neurologist Dr. Charlotte Hollman called it an “opportunity” to diagnose Lexa before birth in order to provide her with care.<\/span><\/p>\n

“We did do an amnio since we knew we had a 1 in 4 chance of having another SMA child,” said Lexa’s mother Andrea James. “It wasn’t as scary this round two. It was just advocating and getting on it. Making sure we had a good path for her.”<\/p>\n

The gene therapy Lexa received, Zolgenesma, is a single-dose medicine administered by IV. It\u2019s for children aged two and under and replaces the missing SM1 gene. The gene then <\/span>produces the required protein<\/span><\/a>, and the disease does not progress any further. Prior to gene therapy being available, there was little doctors could do apart from making patients as comfortable as possible.\u00a0Thanks to prenatal screening<\/a>, doctors were able to secure Zolgenesma to have it ready before Lena was more than a week old.<\/span><\/p>\n

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