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.”

June 08, 2018

Crigler Najjar Symposium


Could Statins Ease Deadly Heart Condition in Rare Neuromuscular Disease?

April 17, 2018

Could Statins Ease Deadly Heart Condition in Rare Neuromuscular Disease?


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James Wilson, MD, PhD, from the University of Pennsylvania, Joins the Rett Syndrome Research Trust’s Gene Therapy Consortium

January 25, 2018

James Wilson, MD, PhD, from the University of Pennsylvania, Joins the Rett Syndrome Research Trust’s Gene Therapy Consortium


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Doctor with rare disease racing to save his own life

January 14, 2018

Doctor with rare disease racing to save his own life


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FAST Summit & Gala Lures 1,300 to Chicago, Raises $4 Million for Angelman Syndrome Research

December 06, 2017

FAST Summit & Gala Lures 1,300 to Chicago, Raises $4 Million for Angelman Syndrome Research


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Healthcare Summit 2017: Spotlight: Treating My Own Disease | Forbes Live

December 01, 2017

Healthcare Summit 2017: Spotlight: Treating My Own Disease | Forbes Live


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CDKL5 Forum

November 29, 2017

CDKL5 Forum


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Gene Therapy Hits a Peculiar Roadblock: A Virus Shortage

November 27, 2017

Gene Therapy Hits a Peculiar Roadblock: A Virus Shortage


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