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Saving Eliza: Gene therapy offers new hope for girl with ‘childhood Alzheimer’s’

Youngster the Epitome of #GeneTherapy's Hope, Promise  #SNRTG #BigData #RareDisease #genomics

  • Eliza received her gene therapy at Nationwide Children’s Hospital in Columbus, Ohio, in May.
  • Eliza is the first patient in the world to get the gene therapy.
  • Saving Eliza: Gene therapy offers new hope for girl with ‘childhood Alzheimer’s’
  • The O’Neills now want to raise more money for other Sanfilippo families to make sure they also have a chance to see their kids take part in clinical trials and improve.
  • As the family hoped and waited for a spot in the trial, Eliza was steadily declining.

Read the full article, click here.


@Primary_Immune1: “Youngster the Epitome of #GeneTherapy’s Hope, Promise #SNRTG #BigData #RareDisease #genomics”


Eliza O’Neill’s parents call it their miracle. She’s finally the first patient to receive an experimental therapy for her deadly disease.


Saving Eliza: Gene therapy offers new hope for girl with ‘childhood Alzheimer’s’

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