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.”

March 19, 2015

Penn and PTC Therapeutics team up to research rare diseases

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March 04, 2015

Gene Therapy's Potential Pricing Problem

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March 03, 2015

Philadelphia Magazine: Spring Charity Bike Rides to Have on Your Radar

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February 27, 2015 World Rare Disease Day: A call for earlier diagnosis and treatment

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February 13, 2015

Penn Orphan Disease Center Announces First Grant Recipients from Million Dollar Bike Ride

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February 06, 2015

Focus on Rare Diseases is Common at Penn Medicine

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January 12, 2015

From the Office of the EVP

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March 06, 2014

$3.25 Million Gift Creates Penn Medicine/CHOP Friedreich's Ataxia Center of Excellence

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