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Jany E. Dagher

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Articles

N -of-1 investigational study of a novel antisense oligonucleotide drug in CHCHD10 -related ALS shows early signs of efficacy

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By Margot A. Cousin, Laurence Mignon, Jaimin Shah, Mercedes Prudencio, Tania F. Gendron, Angita Jain, Evan Udine, Marka van Blitterswijk, Erik H. Middlebrooks, Jens O Watzlawik, Wolfdieter Springer, Tejal Parekh, Zachary T. McEachin, Leonard Petrucelli, Björn Oskarsson, Aditi I. Dagli, Megan H. Donahue, Jany E. Dagher
| Cell Press Verified
Keywords amyotrophic lateral sclerosis antisense oligonucleotide CHCHD10 neurofilament light individualized therapy CAT Scale translation to patients Get full text access Log in, subscribe or purchase for full access. References 1. Bannwarth, S. ∙ Ait-El-Mkadem, S. ∙ Chaussenot, A. ... A mitochondrial origin for frontotemporal dementia and amyotrophic lateral sclerosis through CHCHD10 involvement Brain. 2014; 137:2329-2345 2. Kwan, J.Y. ∙ Lantz, C.I. ∙ Korobeynikov, V.A. ...
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