Study & Trial Learnings to Date – Observations on Design, Endpoints & Patient Enrichment Strategies moderated by Kathie Bishop, PhD, Otonomy.

• Guillaume Bassez, MD, PhD, Institut de Myologie
• Chad Heatwole, MD, University of Rochester
• Joseph Horrigan, MD, AMO Pharma
• Laurence Mignon, PhD, Ionis Pharmaceuticals
• Christopher Turner, MD, PhD, University College London

DM2 State of the Science: Current Research, Next Steps with Allison Formal, MBA.

Current Therapeutic Approaches moderated by Douglas Kerr, MD, PhD, MBA, Generation Bio.

• Andrew Berglund, PhD, RNA Institute
• Vincent Dion, PhD, Cardiff University School of Medicine
• Matthew Disney, PhD, Scripps Research Institute
• Steve Finkbeiner, MD, PhD, Gladstone Institutes
• Darren Monckton, PhD, University of Glasgow
• Christopher Pearson, PhD, Hospital for Sick Children
• Romesh Subramanian, PhD, Dyne Therapeutics
• Eugene Yeo, PhD, UCSD/Locana Bio

DM Animal Models: What We Have, What We Need & Why moderated by Thomas Cooper, MD, Baylor College of Medicine.

• Laurent Bogdanik, PhD, The Jackson Labs
• Genèvieve Gourdon, PhD, Institut de Myologie
• Sharon Hesterlee, PhD, AskBio
• Laura Ranum, PhD, University of Florida
• Ashish Rao, MS, Baylor College of Medicine
• Maurice Swanson, PhD, University of Florida
• Charles Thornton, MD, University of Rochester
• Eric Wang, PhD, University of Florida
• Thurman Wheeler, MD, Massachusetts General Hospital

PRMRP: Specifics of This International Research Funding Program & Strategies for Application with Jonathan Ryder, PhD, US Department of Defense

• Frank Bennett, PhD, Ionis Pharmaceuticals
• Donovan Lott, PhD, PT, University of Florida
• Peg Nopoulos, MD, University of Iowa
• Jeffrey Statland, MD, University of Kansas
• Matthew Tanner, MS, University of Rochester
• Thurman Wheeler, MD, Massachusetts General Hospital