Gene therapy’s new hope: A neuron-targeting virus is saving infant lives

November 01, 2017

CENTREVILLE, VIRGINIA—Nothing unusual jumps out upon meeting Evelyn, a bubbly almost-3-year-old with red curls—except that she should not be here, chatting with a visitor in her family’s living room, twirling in her tights to the Pharrell Williams song “Happy.”

July 26, 2011

Penn Receives $10 Million to Create Center for Orphan Disease Research and Therapy


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July 26, 2011

Penn raises $10 million for 'orphan' disease effort


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Adrenoleukodystrophy Patient Perspective: Turning Despair into a Gene Therapy Breakthrough

April 06, 2011

Adrenoleukodystrophy Patient Perspective: Turning Despair into a Gene Therapy Breakthrough


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December 31, 2010

The Penn Center for Orphan Disease Research & Therapy: Making Rare Disorders a Priority


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