Tag Archive: Katherine High

Dec 21 2017

Groundbreaking Gene Therapy Approved for Inherited Retinal Blindness

More than a decade of studies led by researchers at Children’s Hospital of Philadelphia and the University of Pennsylvania culminated this week with the U.S. Food and Drug Administration’s approval of an innovative one-time gene therapy product indicated for the treatment of patients with a rare, inherited form of retinal blindness.

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Dec 15 2017

Brain Plasticity, Hemophilia B Gene Therapy, Stem Cells and SCD, Medical Cannabis for Autism, PA Rare Disease Advisory Council

The year 2017 might be coming to a close, but research continues to ramp up at Children’s Hospital of Philadelphia, with exciting developments in the fields of brain science, hemophilia, gene therapy, and more. In this week’s roundup of headlines, we take a look at remarkable reports from CHOP and Penn Medicine about the brain’s ability to reorganize itself after limb amputation, the first U.S. effort to observe the use of medical cannabis for children with autism, and exciting innovations to improve sickle cell disease treatment presented at the 59th American Society of Hematology Annual Meeting and Exposition. Read on to discover more about these brilliant breakthroughs. 

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