Empowered Patient Podcast

Dr. Alejandro Dorenbaum is the Chief Medical Officer of Reneo Pharmaceuticals, developing treatments for primary mitochondrial myopathy, a type of rare metabolic muscle disease. Now with genetic testing, there are special panels devoted to diagnosing PMM that can provide quick results for an accurate diagnosis. Unfortunately, no treatment options are currently available, and physicians only treat the complications. REN001, a drug developed by another pharmaceutical company, is being tested in clinical trials to address muscle strength. Alejandro explains, "The myopathies and, specifically, our lead program is in primary mitochondrial myopathy. Primary mitochondrial myopathies are a group of diseases where genes that code for proteins involved in mitochondrial function are affected. So these patients can have defects both in the nuclear genes of the muscle cells or in the mitochondrial genes, and then there are defects in the ability to manage fats in the cell as a source of energy." "We then did a Phase 1 tri