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.”

Piecing Together the Puzzle of a Rare-Among-Rare Bone Disorder

October 09, 2017

Piecing Together the Puzzle of a Rare-Among-Rare Bone Disorder


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FDA Approves Personalized Cellular Therapy for Advanced Leukemia Developed by University of Pennsylvania and Children’s Hospital of Philadelphia

August 30, 2017

FDA Approves Personalized Cellular Therapy for Advanced Leukemia Developed by University of Pennsylvania and Children’s Hospital of Philadelphia


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REGENXBIO Announces IND Active for Phase I Trial of RGX-111 to Treat Mucopolysaccharidosis Type I

August 08, 2017

REGENXBIO Announces IND Active for Phase I Trial of RGX-111 to Treat Mucopolysaccharidosis Type I


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New Biomarker Found for Group of Rare Metabolic Diseases

August 07, 2017

New Biomarker Found for Group of Rare Metabolic Diseases


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Orphan Disease Center brings communities, researchers together to fight rare illnesses

July 15, 2017

Orphan Disease Center brings communities, researchers together to fight rare illnesses


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Penn Orphan Disease Center to Develop Rare Disease Patient Registries

May 31, 2017

Penn Orphan Disease Center to Develop Rare Disease Patient Registries


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Penn Orphan Disease Center Partners with Foundation for Angelman Syndrome Therapeutics on Gene Therapy Research

February 14, 2017

Penn Orphan Disease Center Partners with Foundation for Angelman Syndrome Therapeutics on Gene Therapy Research


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Orphan Disease Center Announces Program of Excellence for Motor Neuron Disease

January 09, 2017

Orphan Disease Center Announces Program of Excellence for Motor Neuron Disease


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