Expert Interview
A Second View: Discussing the potential of sepofarsen ahead of the Illuminate trial readout in LCA10 patients
Ticker(s): PRQRInstitution: University of Miami
- Professor at the Bascom Palmer Eye Institute, University of Miami Miller School of Medicine.
- Clinical scientist with expertise and translational research experience in the areas of hereditary retinal disease, neuro-ophthalmology, and ophthalmic epidemiology.
- Research focus includes gene therapy, clinical visual function testing, ophthalmic molecular imaging, and biomarkers; has participated in numerous clinical trials in hereditary retinal disease and neuro-ophthalmology.
On a scale from 1-10 (10 being extremely excited), where would you rate your level of excitement for sepofarsen?
Added By: c_adminPlease describe your clinical practice; roughly how many patients with LCA10 do you currently manage?
Added By: c_adminwhen we talk about an average response, it is potentially possible that the average or the mean will be kind of influenced significantly by outliers. Let's say that the study is technically successful, but we replicate that heterogeneity again, where there's for example, a 10% or a 15% rate of hyper response, but the vast majority of patients have a fairly modest response. If that drug were approved on the basis of that study, how would you use it?
Added By: c_adminCould the baseline assessments be just unstable? In this case, the baseline was established in the phase 1-2 by doing two assessments and averaging them. And was that enough in order to get a reliable estimate of what the true baseline vision? And if we mis-assigned the original baseline, could some of these deltas that we're seeing on study, not reflect necessarily improvement, but just inaccurately assigned baseline vision?
Added By: c_adminDo you know if ERG was evaluated in these patients?
Added By: c_adminAre You Interested In These Questions?
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