Would I be wrong to assume that Urano is targeting Dantrolene to treat what he perceived as the fundamental defect in Wolframs Syndrome. My assumption is that he can identify people early on with Wolframs and treat with this already available drug.

Also, it is very clear he continues to study MANF and even GC continues to state they have interest in Wolframs. It's a key part of the boiler plate messaging in PR's. My assumption here is that MANF will be used to treat Wolframs induced blindness and aligns very well with the AMBS opthamology portfolio. It is also very clear that MANF is being targeted for regeneration of betas cells in patients that are losing beta cell function due to Wolframs.

To me, Dantrolene, if effective could be used early on in the disease? MANF treats and controls the manifestations of the disease related to blindness, diabetes, and hearing loss.