Kristina Pfau, Josep Callizo, Petra Rossouw, Chrysoula Gabrani, Frank Holz, Peter Charbel Issa, Ulrich Kellner, Rupert Strauss, Laura Kühlewein, Katarina Stingl, Nicolas Feltgen, Maximilian Pfau
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[N-Acetylcysteine (NAC) for Retinitis pigmentosa].
Retinitis pigmentosa (RP) is a genetically mediated degenerative retinal disease that leads to progressive photoreceptor degeneration and, ultimately, blindness. Oxidative stress plays a central role in the pathogenesis of RP. This study examines the effect of N-acetylcysteine (NAC), an antioxidant and glutathione precursor, on the disease progression in RP patients. NAC can reduce oxidative stress in the retina and has shown potential neuroprotective effects in preclinical models. Initial clinical studies indicate improvements in visual acuity and macular function with NAC therapy, though no structural changes have yet been demonstrated. The ongoing multicentre Phase III trial "NAC Attack" aims to evaluate the long-term efficacy and safety of NAC in RP.
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