N-of-1 investigational study of a novel antisense oligonucleotide drug in CHCHD10-related ALS shows early signs of efficacy
Cousin, M.A., Dagli, A.I., Mignon, L., Shah, J.S., Prudencio, M., Gendron, T.F., Jain, A., Udine, E., van Blitterswijk, M., Middlebrooks, E.H., Donahue, M.H., Dagher, J.E., Watzlawik, J.O., Springer, W., Parekh, T., McEachin, Z.T., Petrucelli, L., Oskarsson, B.
