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.”

Skin regeneration with insights

November 08, 2017

Skin regeneration with insights


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Regeneration of the entire human  epidermis using transgenic stem cells

November 08, 2017

Regeneration of the entire human epidermis using transgenic stem cells


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Single-Dose Gene-Replacement Therapy for Spinal Muscular  Atrophy

November 02, 2017

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy


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

November 01, 2017

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


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Could Medical Marijuana Prevent Seizures? Researchers at Penn Medicine Are Hoping to Find Out

October 27, 2017

Could Medical Marijuana Prevent Seizures? Researchers at Penn Medicine Are Hoping to Find Out


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Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy

October 26, 2017

Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy


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Meaningful Data Drives Better Trial Design and Faster Drug Approvals

October 25, 2017

Meaningful Data Drives Better Trial Design and Faster Drug Approvals


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FDA Advisory Committee Unanimously Recommends Approval of Investigational LUXTURNA™ (voretigene neparvovec) for Patients with Biallelic RPE65-mediated Inherited Retinal Disease

October 12, 2017

FDA Advisory Committee Unanimously Recommends Approval of Investigational LUXTURNA™ (voretigene neparvovec) for Patients with Biallelic RPE65-mediated Inherited Retinal Disease


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