Delivering RNA Therapeutics for FSHD: Innovations in DUX4 Silencing & Biomarkers for Clinical Development

• Exploring the use of RNA therapeutics for the treatment of FSHD to deliver efficacious patient outcomes
• Uncovering novel DUX4 silencing technologies and biomarkers to tackle challenges with patient heterogeneity and asymmetric disease progression
• Strengthening end point selection to reduce trial uncertainty and increase confidence in development decisions

Speakers:

Thomas Natoli

Senior Director, Neuromuscular Research

Dyne Therapeutics