children with inherited mitochondrial disease; leiomyosarcoma; acute myelogenous leukemia; and aromatic L-amino acid decarboxylase deficiency. PTC Therapeutics works in collaboration with various ...
PTC has "pioneered a new approach to treating highly morbid neurologic diseases," said Matthew Klein, the company's chief executive. "I am proud of our team's unwavering commitment to achieve this ...
Alltrna, a Flagship Pioneering company unlocking transfer RNA (tRNA) biology and pioneering tRNA therapeutics to regulate the protein universe and resolve disease, today announced the presentation of ...
It was also under development for the treatment of Rett syndrome, Melas syndrome, Leber’s hereditary optic neuropathy and mitochondrial diseases such as Pearson syndrome, a form of mitochondrial ...