Discovering an Innovative Approach to Trial Design for Real World SMA Population

Time: 1:50 pm
day: Day One


• 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 to the efficacy and building efficacy end-points into both stages

• How submitting PKPD and exploratory efficacy data early on led to seamless development, phase 1 to phase 3 and fast regulatory approval, filling for the FDA with early data