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Comment
. 2020 Sep;16(9):460.
doi: 10.1038/s41582-020-0393-8.

Moving towards genomic therapy for amyotrophic lateral sclerosis

Heather Wood  1
Affiliations
Comment

Moving towards genomic therapy for amyotrophic lateral sclerosis

Heather Wood. Nat Rev Neurol. 2020 Sep.
No abstract available

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Comment on

  • Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS.
    Miller T, Cudkowicz M, Shaw PJ, Andersen PM, Atassi N, Bucelli RC, Genge A, Glass J, Ladha S, Ludolph AL, Maragakis NJ, McDermott CJ, Pestronk A, Ravits J, Salachas F, Trudell R, Van Damme P, Zinman L, Bennett CF, Lane R, Sandrock A, Runz H, Graham D, Houshyar H, McCampbell A, Nestorov I, Chang I, McNeill M, Fanning L, Fradette S, Ferguson TA. Miller T, et al. N Engl J Med. 2020 Jul 9;383(2):109-119. doi: 10.1056/NEJMoa2003715. N Engl J Med. 2020. PMID: 32640130 Clinical Trial.
  • SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS.
    Mueller C, Berry JD, McKenna-Yasek DM, Gernoux G, Owegi MA, Pothier LM, Douthwright CL, Gelevski D, Luppino SD, Blackwood M, Wightman NS, Oakley DH, Frosch MP, Flotte TR, Cudkowicz ME, Brown RH Jr. Mueller C, et al. N Engl J Med. 2020 Jul 9;383(2):151-158. doi: 10.1056/NEJMoa2005056. N Engl J Med. 2020. PMID: 32640133 Free PMC article.

References

Original articles
    1. Miller, T. et al. Phase 1–2 trial of antisense oligonucleotide tofersen for SOD1 ALS. N. Engl. J. Med. 383, 109–119 (2020) - DOI
    1. Mueller, C. et al. SOD1 suppression with adeno-associated virus and microRNA in familial ALS. N. Engl. J. Med. 383, 151–158 (2020) - DOI

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