As Blindness Gene Therapy Nears the FDA Finish Line, A Shout-Out to Activist Families
On a spectacular September Sunday in 2008, 8-year-old Corey Haas, using his cane and holding his mother�s hand, stepped tentatively forward on the pathway leading into the Philadelphia zoo. Hearing kids yelling about the giant balloon hanging above the zoo, he looked up � and screamed. It was the first time he�d seen the sun. Corey was headed toward certain blindness when he�d had gene therapy at Children�s Hospital of Philadelphia, just days earlier.
So begins the talk I�ve given many times since publication of my book, "The Forever Fix: Gene Therapy and the Boy Who Saved It.
Last Thursday, I was glued to my laptop, watching and listening to physicians, researchers, family, and patients present their cases for FDA approval of
Luxturna (voretigene neparvovec), the gene therapy that Corey, now a high school senior, received. He and dozens of others participating in several clinical trials can now see, thanks to the gene therapy for RPE65-mediated inherited retinal dystrophy. The treatment introduces functioning genes into the thin layer of pigmented cells that hugs the rods and cones � one time.
So begins the talk I�ve given many times since publication of my book, "The Forever Fix: Gene Therapy and the Boy Who Saved It.
Last Thursday, I was glued to my laptop, watching and listening to physicians, researchers, family, and patients present their cases for FDA approval of
Luxturna (voretigene neparvovec), the gene therapy that Corey, now a high school senior, received. He and dozens of others participating in several clinical trials can now see, thanks to the gene therapy for RPE65-mediated inherited retinal dystrophy. The treatment introduces functioning genes into the thin layer of pigmented cells that hugs the rods and cones � one time.
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