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Penn Study Suggests Genetic Disease CDKL5 Deficiency Disorder Could Be Treatable after Childhood

Future of Rare Disease Research: Ethics, Treatments, and Access

Date Time Future of Rare Disease Research: Ethics, Treatments, and Access PHILADELPHIA- In celebration of the 10th Anniversary of the Orphan Disease Center, pioneering gene therapy scientist James Wilson, MD, PhD, a professor and director of the Penn Gene Therapy Program and the Penn Orphan Disease Center at the Perelman School of Medicine (PSOM) at the University of Pennsylvania, will lead a two-day symposium, featuring former Prime Minister of the United Kingdom and rare disease parent David Cameron and National Institutes of Health Director Francis Collins, MD, PhD, among many others. The event will take place on Wednesday, February 24th and Thursday, February 25th.

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