Foundation Fighting Blindness

News from the Foundation

Latest Update: Gene Therapy Restores Significant Vision to Children with LCA

Gene therapy has taken another major step forward as a treatment for retinal diseases. Researchers conducting the landmark gene therapy clinical trial at The Children’s Hospital of Philadelphia (CHOP) have just reported that 12 people with Leber congenital amaurosis have now been successfully treated and are experiencing sustained, and in many cases significant, improvement in their vision.
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Latest Update: Childhood Rediscovered: Gene Therapy Restores Vision for 9-year old with LCA

When it was his turn to take the stage at a press conference at The Children’s Hospital of Philadelphia (CHOP), Corey Haas got up from his seat in the auditorium and walked effortlessly up the four steps in front of him. Without hesitation, he crossed the stage, stepped around the table and took his seat next to Dr. Jean Bennett. His white cane was nowhere to be seen.
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New LCA Gene Therapy Clinical Trial Recruiting Participants

Applied Genetic Technologies Corporation (AGTC) has launched a Phase I/II clinical trial of gene therapy to treat Leber congenital amaurosis, extending the work being done for similar treatments in three other Phase I clinical trials taking place at The Children’s Hospital of Philadelphia, Universities of Pennsylvania and Florida, and Moorfield’s Eye Hospital in the U.K. In this trial, AGTC will be evaluating higher doses of the treatment in younger participants with the hope of restoring even more vision than has been restored in the other three studies.

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