Paulo Fontoura
Company: Roche
Job title: Global Head & SVP, Neuroscience & Rare Diseases Clinical Development
Seminars:
Live Q&A 2:50 pm
day: Day One
Discovering an Innovative Approach to Trial Design for Real World SMA Population 1:50 pm
• Designing a clinical development program across a range of ages and disabilities • Evaluating whether SMN protein or other blood biomarker levels associate with improved motor function in SMA patients and using these to monitor or predict treatment outcomes • Determining the right dose based on the biomarker, pivot from dose ranging safety portion…Read more
day: Day One
Kick-Off Panel Discussion: Dissecting Fundamental Questions Arising for Each Therapeutic Approach 9:10 am
• A critical overview of the current status of knowledge and therapies for neuromuscular disorders, focusing on ALS, SMA and DMD • What have we unravelled over the last year that will drive more effective clinical development to reach patients in need? • What are the unique challenges for ASO therapy, gene therapy, gene modification…Read more
day: Day One