Soraprazan – a new regenerative therapy for Stargardt’s disease
Abnormal accumulation of lipofuscin in the RPE cells is a hallmark in Stargardt's disease. As lipofuscin cannot be removed from the RPE cells in the eyes neither spontaneously nor with existing therapies, the accumulation of lipofuscin in the RPE cells of the eyes results in the degradation of the RPE cells and consequently in worsening of visual acuity and could lead to blindness. Soraprazan was able to show removal of lipofuscin in RPE cells. Aim of the phase II trial in this project is to evaluate savety and efficacy or orally taken Soraprazan treatment compared to placebo.
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Funding SchemeRIA - Research and Innovation action