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Understanding Spark's LUXTURNA potential in RPE65-mediated IRD blindness currently under review at the FDATicker(s): ONCE
An Ophthalmologist who treats pediatric patients with IRD. Ideally this expert would have participated in a developmental trial for LUXTURNA.
Please describe your clinical practice. How many patients do you treat with IRD, how are they managed currently?Added By: joe_mccann
Can we walk through the primary endpoint of bilateral multi-luminance mobility testing (MLMT) from the Lancet study? How well established is this endpoint and how consistent is the decline among untreated patients?Added By: joe_mccann
LUXTURNA showed a marked difference compared to control participants across the first two secondary endpoints: full-field light sensitivity threshold (FST) testing (p<0.001) and the mobility test change score for the first injected eye (p=0.001). A third secondary endpoint, the change in visual acuity (VA) averaged over both eyes, was not statistically significant between intervention and control participants (p=0.17).
Let's discuss the 3 endpoints and how concerning it is the drug didn't hit a statistically significant benefit on the VA endpoint.
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