PRESENTATION TITLE
Piloting Pathways for “Interventional Genomics” in Orphan Neurologic Disease

PRESENTER(S)
Timothy Wei-Wen Yu, MD, PhD

PRESENTER BIO
Dr. Yu is a neurogeneticist at Boston Children’s Hospital and Harvard Medical School. He obtained his undergraduate degree in Biochemistry at Harvard College, which inspired an early preoccupation with structural biology and rational drug design. Those interests shifted during his MD-PhD training at UCSF in which he focused on neuroscience and C. elegans genetics. After completing neurology residency at Massachusetts General Hospital and Brigham and Women’s Hospital, he joined the faculty at Boston Children’s Hospital, where he has found his work to have come full circle, combining human genetics, neuroscience, and molecular medicine.

He currently serves as a Staff Physician and Researcher in the Division of Genetics and Genomics and the Department of Neurology at BCH, Associate Professor at Harvard Medical School and an Associate Member of the Broad Institute.

LEARNING OBJECTIVES 
After the presentation, attendees will be able to:

• Recognize key genetic and clinical criteria by which candidates for investigational “n of 1” antisense oligonucleotide intervention are being selected
• Describe clinical risks of antisense oligonucleotide administration
• Describe the evolving landscape of stakeholders in the US and abroad invested in studying the safety, efficacy, and scalability of “n of 1” treatments, and the key opportunities and challenges they are addressing

ATTENDANCE / CREDIT
Text the session code (provided only at the session) to 507-200-3010 within 48 hours of the live presentation to record attendance. All learners are encouraged to text attendance regardless of credit needs. This number is only used for receiving text messages related to tracking attendance. Additional tasks to obtain credit may be required based on the specific activity requirements and will be announced accordingly. Swiping your badge will not provide credit; that process is only applicable to meet GME requirements for Residents & Fellows.

TRANSCRIPT
Any credit or attendance awarded from this session will appear on your Transcript.

For disclosure information regarding Mayo Clinic School of Continuous Professional Development accreditation review committee member(s) and staff, please go here to review disclosures.

QUESTIONS?
Contact Coordinator Chris Scott 507-284-0426