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Comment
. 2018 Jan;14(1):1.
doi: 10.1038/nrneurol.2017.163. Epub 2017 Nov 17.

Motor neuron disease: Positive trial results published for ground-breaking SMA therapies

Charlotte Ridler
Comment

Motor neuron disease: Positive trial results published for ground-breaking SMA therapies

Charlotte Ridler. Nat Rev Neurol. 2018 Jan.
No abstract available

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  • Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.
    Mendell JR, Al-Zaidy S, Shell R, Arnold WD, Rodino-Klapac LR, Prior TW, Lowes L, Alfano L, Berry K, Church K, Kissel JT, Nagendran S, L'Italien J, Sproule DM, Wells C, Cardenas JA, Heitzer MD, Kaspar A, Corcoran S, Braun L, Likhite S, Miranda C, Meyer K, Foust KD, Burghes AHM, Kaspar BK. Mendell JR, et al. N Engl J Med. 2017 Nov 2;377(18):1713-1722. doi: 10.1056/NEJMoa1706198. N Engl J Med. 2017. PMID: 29091557 Clinical Trial.
  • Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy.
    Finkel RS, Mercuri E, Darras BT, Connolly AM, Kuntz NL, Kirschner J, Chiriboga CA, Saito K, Servais L, Tizzano E, Topaloglu H, Tulinius M, Montes J, Glanzman AM, Bishop K, Zhong ZJ, Gheuens S, Bennett CF, Schneider E, Farwell W, De Vivo DC; ENDEAR Study Group. Finkel RS, et al. N Engl J Med. 2017 Nov 2;377(18):1723-1732. doi: 10.1056/NEJMoa1702752. N Engl J Med. 2017. PMID: 29091570 Clinical Trial.

References

    1. N Engl J Med. 2017 Nov 2;377(18):1713-1722 - PubMed
    1. N Engl J Med. 2017 Nov 2;377(18):1723-1732 - PubMed

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