(0)

Already a DIA Member? Sign in. Not a member? Join.

Sign in

Forgot User ID? or Forgot Password?

Not a Member?

Create Account and Join

Hyatt Regency Bethesda

Oct 28, 2019 7:00 AM - Oct 29, 2019 12:15 PM

One Bethesda Metro Center, 7400 Wisconsin Avenue, Bethesda, MD 20814-5326

Pediatric Drug Development Workshop

Back to Agenda

Session 1: Developing Innovative Technologies for Children: Pediatric-Directed Drug Development

Session Chair(s)

Andrew Mulberg, MD

Andrew Mulberg, MD

Senior Vice President

Neurogene, United States

This session will explore the innovative use of technologies in pediatric drug development. A presentation of scientific underpinnings of novel technology for the treatment of inborn errors of metabolism and CNS neurodegenerative diseases will be presented. Overcoming the technical challenges of gene therapy to the CNS, approaches for improved protein expression targeting, and stability for novel gene therapy. The presentations will be followed by a panel discussion.

Learning Objective :

At the conclusion of this session, participants should be able to:

  • understand the science and technology of novel gene transfer and therapies on evolution of potential cures for treatment of neurodegenerative diseases affecting children
  • develop an appreciation and understanding of the science underlying novel approaches to gene transfer in inborn errors of metabolism

Speaker(s)

James M Wilson, MD, PhD

Speaker

James M Wilson, MD, PhD

University of Pennsylvania, United States

Kathrin Meyer, DrSc, PhD

Speaker

Kathrin Meyer, DrSc, PhD

Nationwide Children’s Hospital, United States

Principal Investigator, Assistant Professor, The Ohio State University

Cheryl Yoder, RN

Panelist

Cheryl Yoder, RN

Cure SMA, United States

Patient Advocate

Have an account?

Be informed and stay engaged.

Don't miss an opportunity - join our mailing list to stay up to date on DIA insights and events.