{"page":"\u003clink rel=\"stylesheet\" href=\"https://lessonplanet.com/assets/packs/css/resources-c03aa079.css\" /\u003e\n\u003clink rel=\"stylesheet\" href=\"https://lessonplanet.com/assets/packs/css/lp_boclips_stylesheets-517835be.css\" media=\"all\" /\u003e\n\u003cdiv data-title='Baby gene therapy study offers hope for fatal muscle disease ++Animation mute++' data-url='/boclips/videos/5c54d666d8eafeecae203509' data-video-url='/boclips/videos/5c54d666d8eafeecae203509' id='bo_player_modal'\u003e\n\u003cdiv class='boclips-resource-page modal-dialog panel-container'\u003e\n\u003cdiv class='react-notifications-root'\u003e\u003c/div\u003e\n\u003cdiv class='rp-header'\u003e\n\u003cdiv class='rp-type'\u003e\n\u003ci aria-hidden='true' class='fai fa-regular fa-circle-play'\u003e\u003c/i\u003e\nVideo\n\u003c/div\u003e\n\u003ch1 class='rp-title' id='video-title'\u003e\nBaby gene therapy study offers hope for fatal muscle disease ++Animation mute++\n\u003c/h1\u003e\n\u003cdiv class='rp-actions'\u003e\n\u003cdiv class='mr-1'\u003e\n\u003ca class=\"btn btn-success\" data-posthog-event=\"Signup: LP Signup Activity\" data-posthog-location=\"body_link_boclips\" data-remote=\"true\" href=\"/subscription/new\"\u003e\u003cspan\u003e\u003cspan\u003eGet Free Access\u003c/span\u003e\u003cspan class=\"\"\u003e for 10 Days\u003c/span\u003e\u003cspan\u003e!\u003c/span\u003e\u003c/span\u003e\u003c/a\u003e\n\u003c/div\u003e\n\u003c/div\u003e\n\u003c/div\u003e\n\u003cdiv class='rp-body'\u003e\n\u003cdiv class='rp-info'\u003e\n\u003cdiv aria-label='Hide resource details' class='rp-hide-info' role='button' tabindex='0'\u003e\u0026times;\u003c/div\u003e\n\u003ci aria-label='Expand resource details' class='rp-expand-info fai fa-solid fa-up-right-and-down-left-from-center' role='button' tabindex='0'\u003e\u003c/i\u003e\n\u003ci aria-label='Compress resource details' class='rp-compress-info fai fa-solid fa-down-left-and-up-right-to-center' role='button' tabindex='0'\u003e\u003c/i\u003e\n\u003cdiv class='rp-rating'\u003e\n\u003cspan class='resource-pool'\u003e\n\u003cspan class='pool-label'\u003ePublisher:\u003c/span\u003e\n\u003cspan class='pool-name'\u003e\n\u003cspan class='text'\u003e\u003ca data-publisher-id=\"30356011\" href=\"/search?publisher_ids%5B%5D=30356011\"\u003eCurated Video\u003c/a\u003e\u003c/span\u003e\n\u003c/span\u003e\n\u003c/span\u003e\n\u003c/div\u003e\n\u003cdiv class='rp-description'\u003e\n\u003cspan class='short-description'\u003eLEAD IN:               Researchers are reporting a gene therapy has been used for the first time to help babies with a muscle disease that leaves them unable to move, swallow and breathe.              They say it's extending the infants'...\u003c/span\u003e\n\u003cspan class='full-description hide'\u003eLEAD IN: \u003cbr/\u003e              Researchers are reporting a gene therapy has been used for the first time to help babies with a muscle disease that leaves them unable to move, swallow and breathe.\u003cbr/\u003e              They say it's extending the infants' lives.\u003cbr/\u003e              \u003cbr/\u003e              STORY-LINE:\u003cbr/\u003e              A first attempt at gene therapy for a disease that leaves babies unable to move, swallow and, eventually, breathe has extended the tots' lives, and some began to roll over, sit and stand on their own, researchers say ( Wednesday 2 November 2017).\u003cbr/\u003e              Only 15 babies with spinal muscular atrophy received the experimental gene therapy, but researchers in Ohio credited the preliminary and promising results to replacing the infants' defective gene early — in the first few months of life, before the neuromuscular disease destroyed too many key nerve cells.\u003cbr/\u003e              Spinal muscular atrophy occurs in about 1 in 10,000 births, and those with the most severe form, called SMA Type 1, rarely reach their second birthday. They can be born looking healthy but rapidly decline. One study found just 8 percent of the most \u003cbr/\u003e              severely affected survived to age 20 months without needing permanent mechanical ventilation to breathe.\u003cbr/\u003e              There is no cure. The first treatment wasn't approved until last December — a drug named Spinraza that requires spinal injections every few months.\u003cbr/\u003e              The experimental gene therapy approach aims for a one-time fix.\u003cbr/\u003e              WHAT GOES WRONG\u003cbr/\u003e              Spinal muscular atrophy is caused when a mutated gene can't produce a protein crucial for survival of motor neurons, nerve cells in the spinal cord that control muscles.\u003cbr/\u003e              Some children carry extra copies of a backup gene that produces small amounts of the vital protein, and thus have much milder forms of the disease.\u003cbr/\u003e              GENE REPLACEMENT\u003cbr/\u003e              Scientists loaded a healthy version of the gene into a virus modified so it couldn't cause illness. Then 15 babies got a one-time intravenous injection. The virus carried the healthy gene into motor neurons, where it got to work producing the protein those \u003cbr/\u003e              nerve cells require to live.\u003cbr/\u003e              Three babies received a low dose of the gene therapy, as a first-step safety precaution. The remaining 12 got a high dose.\u003cbr/\u003e              RESULTS\u003cbr/\u003e              All of the children are alive, Mendell said, about two years and counting after treatment. All beat the odds of needing permanent machine help to breathe by age 20 months.\u003cbr/\u003e              But only the high-dose recipients saw better motor control, reaching some developmental milestones usually unthinkable for these patients. Eleven could sit unassisted at least briefly; nine could roll over. Eleven are speaking and able to swallow. Two were able to crawl, stand and then walk.\u003cbr/\u003e              The only serious side effect attributed to the gene therapy so far involved possible signs of a liver problem that eased with treatment.\u003cbr/\u003e              NEXT STEPS\u003cbr/\u003e              AveXis Inc., which is developing the gene therapy and helped fund Wednesday's study, has opened a second small trial at seven hospitals.\u003cbr/\u003e              Meanwhile, doctors are prescribing SMA patients the new medication Spinraza, which works by increasing that backup gene's protein production and, according to a separate New England Journal study, had some benefit in about half of patients. The first \u003cbr/\u003e              year of treatment costs about $750,000, an accompanying editorial noted.\u003cbr/\u003e              With the drug's availability, some health groups are urging that SMA be added to the list of diseases that all newborns are screened for, so parents can seek early treatment.\u003cbr/\u003e              =========\u003cbr/\u003e              This Associated Press series was produced in partnership with the Howard Hughes Medical Institute's Department of Science Education. The AP is solely responsible for all content.\u003cbr/\u003e            \u003cbr/\u003e\u003cbr/\u003e              New York - 1 November 2017\u003cbr/\u003e              1. 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