Ernest Balaguer on Muck Rack

Ernest Balaguer

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Articles

LRRK2 -targeting antisense oligonucleotide in Parkinson’s disease: a phase 1 randomized controlled trial

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By Omar S. Mabrouk, Ben Tichler, Eva Thoma, Sara Alexanian, Jingxian Chen, Tzu-Ying Liu, John Annand, Unnati Kapadnis, Kyle Ferber, Yuka Moroishi, Julie M Czerkowicz, Jennifer A. Inra, Ronald B. Postuma, Tanya Gurevich, Pablo Mir, Huw Morris, Jason Aldred, Matthew Brodsky, Aaron Ellenbogen, Christopher M Tolleson, Andrew Siderowf, Charalampos Tzoulis, María Martí, Hien T. Zhao, Holly B. Kordasiewicz, Roger M Lane, Warren D. Hirst, Stephanie Fradette, Danielle Graham, H. Moore Arnold, Beth Hirschhorn, Roy Llorens Arenas, Alan A. Shomo, Kelly E. Glajch, Danielle Larson, Ernest Balaguer
| Nature Verified
Abstract LRRK2 (encoding leucine-rich repeat kinase 2) variants are the most common genetic cause of Parkinson’s disease (PD). Lowering LRRK2 levels and/or inhibiting LRRK2 activity may modify PD-associated neuropathology. BIIB094 (ION859), an antisense oligonucleotide, targets LRRK2 mRNA for degradation. REASON was a first-in-human randomized phase 1 study investigating the safety, tolerability, pharmacokinetics and pharmacodynamics of intrathecal BIIB094 in patients with PD.
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