Events
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Week of Events
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N=1 Collaborative Seminar – Project Butterfly: from drug development to clinical trial. A personalized antisense oligonucleotide medicine for a rare form of CLN3 Batten Disease
N=1 Collaborative Seminar – Project Butterfly: from drug development to clinical trial. A personalized antisense oligonucleotide medicine for a rare form of CLN3 Batten Disease
The presentation will review our development of a personalized antisense oligonucleotide (ASO) medicine for two children with CLN3 Batten disease. We will detail the collaborative process of designing the ASO therapy, achieving FDA IND approval, and establishing a clinical trial protocol.
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Grand Rounds
Grand Rounds
The School of Medicine of The University of North Carolina at Chapel Hill is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.