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Life-Saving Treatments for Spinal Muscular Atrophy: Global Access and Availability.
Armengol VD, Darras BT, Abulaban AA, Alshehri A, Barisic N, Ben-Omran T, Bernert G, Castiglioni C, Chien YH, Farrar MA, Kandawasvika G, Khadilkar S, Mah J, Marini-Bettolo C, Osredkar D, Pfeffer G, Piazzon FB, Pitarch Castellano I, Quijano-Roy S, Saito K, Shin JH, Vázquez-Costa JF, Walter MC, Wanigasinghe J, Xiong H, Griggs RC, Roy B. Armengol VD, et al. Among authors: farrar ma. Neurol Clin Pract. 2024 Feb;14(1):e200224. doi: 10.1212/CPJ.0000000000200224. Epub 2023 Dec 15. Neurol Clin Pract. 2024. PMID: 38107546
Spinal muscular atrophy: molecular mechanisms.
Farrar MA, Johnston HM, Grattan-Smith P, Turner A, Kiernan MC. Farrar MA, et al. Curr Mol Med. 2009 Sep;9(7):851-62. doi: 10.2174/156652409789105516. Curr Mol Med. 2009. PMID: 19860664 Review.
Emerging therapies and challenges in spinal muscular atrophy.
Farrar MA, Park SB, Vucic S, Carey KA, Turner BJ, Gillingwater TH, Swoboda KJ, Kiernan MC. Farrar MA, et al. Ann Neurol. 2017 Mar;81(3):355-368. doi: 10.1002/ana.24864. Epub 2017 Feb 17. Ann Neurol. 2017. PMID: 28026041 Free PMC article. Review.
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 Free article. Clinical Trial.
New and developing therapies in spinal muscular atrophy.
Kariyawasam D, Carey KA, Jones KJ, Farrar MA. Kariyawasam D, et al. Among authors: farrar ma. Paediatr Respir Rev. 2018 Sep;28:3-10. doi: 10.1016/j.prrv.2018.03.003. Epub 2018 Apr 5. Paediatr Respir Rev. 2018. PMID: 29703692 Review.
215 results