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CNN CNN This Morning June 4, 2024 12:27:00

Last week the agency s leader testified to a senate committee. we agree that this is an area we ve got to move along more quickly. reporter: as forc saoirse, next month she will get the gene therapy. she had a preparatory visit earlier this month. her family looking forward to the day she like other 5-year-olds. she is excited to go to school and wants to go to a dodger game and she is inviting everybody to disney world for her. reporter: after years of waiting, saoirse and her family, thrilled for the day she can finally get out into the world. now, she is aware that she has a long hospital stay coming up filled with pokes and shots, but she says she is ready because she knows that s what s going to get her to disney world, to the dodgers game and to kindergarten. yeah, anything to get to ....

The Agency , Gene Therapy , Forc Saoirse , Dodger Game , Disney World , Hospital Stay ,

CNN CNN This Morning June 4, 2024 12:25:00

But didn t go trick-or-treating like other children you. any germ, even a common cold, could kill her because she was born with severe combined immunodeficiency, or bubble boy disease. made famous by the 1976 john travolta movie the boy in the plastic bubble. she was born in hawaii where her father was stationed when she was six days old she was airlifted to ucla mattel children s hospital where she spent two months. for the first year of her life she never left her bedroom. reporter: her first christmas gifts wiped clean and brought to her room. later medications helped. six to seven shots a months. reporter: and she could go outside, but still not near anyone except her immediate family. then dr. cone, who runs this lab at ucla, had good news for the family. he said it looked like in the not too distant future saoirse would be able to get a treatment called gene therapy. he worked on it for nearly 40 ....

Common Cold , Bubble Boy Disease , Didn T Go Trick Or Treating , John Travolta , Ucla Mattel , The Boy In Plastic Bubble , Children S Hospital , Gene Therapy ,