Nano-rare Patient Colloquium 2026 – Recap
October 7-8, 2026 - Cambridge, MA Join us for our annual Nano-rare patient Colloquium. This event is free to attend — register today!
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.
Sneak Peek of the 2026 Nano-rare Patient Colloquium
Brady Huggett interviews Stan Crooke
Realities of the Nano-rare: Siblings, Unfairness and Hard Truths
with Sally Jackson
Silencing ALS
with Neil Shneider, M.D., Ph.D.
Individualized antisense oligonucleotides for SCN2A-related developmental epileptic encephalopathy
Kim-McManus, O., Mignon, L., Douville, J., Pu, H., Parisien, C., Glass, S., Bennett, C.F., Celso, J., Robbins, K., Ung, H., Olson, H., Kingsmore, S.F., Petrou, S., Crooke, S.T., Gleeson, J.G., Berry-Kravis, E. Download PDF
100th Episode: The Personal Rare Disease Journey Behind CNBC Cures
with Becky Quick
