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Clinical Trials Corner: September 2017. J Huntingtons Dis 2017; 6: 255\u201363.","DOI":"10.3233\/JHD-170262"},{"key":"10.3233\/JHD-229002_ref7","doi-asserted-by":"crossref","unstructured":"Palacino J, Swalley SE, Song C, et al. SMN2 splice modulators enhance U1\u2013pre-mRNA association and rescue SMA mice. Nat Chem Biol 2015; 11: 511\u20137.","DOI":"10.1038\/nchembio.1837"},{"key":"10.3233\/JHD-229002_ref8","unstructured":"Sivasankaran R. From spinal muscular atrophy to Huntington\u2019s disease therapeutics. CHDI Foundation Annual Therapeutics Conference 27-29th April 2021."},{"key":"10.3233\/JHD-229002_ref9","doi-asserted-by":"crossref","unstructured":"Gubser Keller C, Shin Y, Monteys AM, et al. An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion. 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Huntingtin Haplotypes Provide Prioritized Target Panels for Allele-specific Silencing in Huntington Disease Patients of European Ancestry. Mol Ther 2015; 23: 1759\u201371.","DOI":"10.1038\/mt.2015.128"},{"key":"10.3233\/JHD-229002_ref14","unstructured":"Schobel SA. Preliminary results from a 15-month open-label extension study investigating tominersen (RG6042) huntingtin protein antisense oligonucleotide in adults with manifest Huntington\u2019s disease. CHDI Foundation Annual Therapeutics Conference 24\u201327th February 2020."},{"key":"10.3233\/JHD-229002_ref15","doi-asserted-by":"crossref","unstructured":"Schobel S, Palermo G, Trundell D, et al. A Global Development Program Testing RG6042, an Antisense Oligonucleotide, for the Treatment of Early Manifest Huntington\u2019s Disease. European Huntington\u2019s Disease Network 2018 Plenary Meeting. Vienna, Austria, 2018.","DOI":"10.1136\/jnnp-2018-EHDN.262"},{"key":"10.3233\/JHD-229002_ref16","unstructured":"Schobel SA. 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