Patient-derived mini brains reveal mechanism and treatment for rare genetic disease Dehydrodolichyl diphosphate synthase (DHDSS)-related disease has no available treatments. The new work could finally change that with nicotinamide mononucleotide (NMN). Written byAllison Whitten, PhD

https://www.drugdiscoverynews.com/patient-derived-mini-brains-reveal-mechanism-and-treatment-for-rare-genetic-disease-17282 Last week at the annual conference of the European Society of Human Genetics in Gothenburg, Sweden, researchers presented work on the first cortical brain organoid models of dehydrodolichyl diphosphate synthase (DHDSS)-related disease — a condition with no current treatments. The pioneering work, led by Eva Morava and Tamas Kozicz's team at the Icahn School of Medicine at Mount Sinai, allowed the scientists to not only elucidate the mechanism of the disease, but also test out a widely available therapeutic — nicotinamide mononucleotide (NMN), better known as vitamin B3.