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Natural history of Type 2 and 3 spinal muscular atrophy: 2-year NatHis-SMA study.
Annoussamy M, Seferian AM, Daron A, Péréon Y, Cances C, Vuillerot C, De Waele L, Laugel V, Schara U, Gidaro T, Lilien C, Hogrel JY, Carlier P, Fournier E, Lowes L, Gorni K, Ly-Le Moal M, Hellbach N, Seabrook T, Czech C, Hermosilla R, Servais L; NatHis-SMA study group. Annoussamy M, et al. Among authors: gorni k. Ann Clin Transl Neurol. 2021 Feb;8(2):359-373. doi: 10.1002/acn3.51281. Epub 2020 Dec 24. Ann Clin Transl Neurol. 2021. PMID: 33369268 Free PMC article.
Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial.
Bertini E, Dessaud E, Mercuri E, Muntoni F, Kirschner J, Reid C, Lusakowska A, Comi GP, Cuisset JM, Abitbol JL, Scherrer B, Ducray PS, Buchbjerg J, Vianna E, van der Pol WL, Vuillerot C, Blaettler T, Fontoura P; Olesoxime SMA Phase 2 Study Investigators. Bertini E, et al. Lancet Neurol. 2017 Jul;16(7):513-522. doi: 10.1016/S1474-4422(17)30085-6. Epub 2017 Apr 28. Lancet Neurol. 2017. PMID: 28460889 Free article. Clinical Trial.
Risdiplam treatment has not led to retinal toxicity in patients with spinal muscular atrophy.
Sergott RC, Amorelli GM, Baranello G, Barreau E, Beres S, Kane S, Mercuri E, Orazi L, SantaMaria M, Tremolada G, Santarsiero D, Waskowska A, Yashiro S, Denk N, Fürst-Recktenwald S, Gerber M, Gorni K, Jaber B, Jacobsen B, Mueller L, Nave S, Scalco RS, Marzoli SB; FIREFISH, SUNFISH, JEWELFISH Working Groups. Sergott RC, et al. Among authors: gorni k. Ann Clin Transl Neurol. 2021 Jan;8(1):54-65. doi: 10.1002/acn3.51239. Epub 2020 Nov 24. Ann Clin Transl Neurol. 2021. PMID: 33231373 Free PMC article.
Long-term follow-up of patients with type 2 and non-ambulant type 3 spinal muscular atrophy (SMA) treated with olesoxime in the OLEOS trial.
Muntoni F, Bertini E, Comi G, Kirschner J, Lusakowska A, Mercuri E, Scoto M, van der Pol WL, Vuillerot C, Burdeska A, El-Khairi M, Fontoura P, Ives J, Gorni K, Reid C, Fuerst-Recktenwald S; OLEOS Study Group. Muntoni F, et al. Among authors: gorni k. Neuromuscul Disord. 2020 Dec;30(12):959-969. doi: 10.1016/j.nmd.2020.10.008. Epub 2020 Nov 5. Neuromuscul Disord. 2020. PMID: 33246887 Free article. Clinical Trial.
Risdiplam in Type 1 Spinal Muscular Atrophy.
Baranello G, Darras BT, Day JW, Deconinck N, Klein A, Masson R, Mercuri E, Rose K, El-Khairi M, Gerber M, Gorni K, Khwaja O, Kletzl H, Scalco RS, Seabrook T, Fontoura P, Servais L; FIREFISH Working Group. Baranello G, et al. Among authors: gorni k. N Engl J Med. 2021 Mar 11;384(10):915-923. doi: 10.1056/NEJMoa2009965. Epub 2021 Feb 24. N Engl J Med. 2021. PMID: 33626251 Clinical Trial.
Prognostic Factors and Treatment-Effect Modifiers in Spinal Muscular Atrophy.
Baranello G, Gorni K, Daigl M, Kotzeva A, Evans R, Hawkins N, Scott DA, Mahajan A, Muntoni F, Servais L. Baranello G, et al. Among authors: gorni k. Clin Pharmacol Ther. 2021 Dec;110(6):1435-1454. doi: 10.1002/cpt.2247. Epub 2021 Jun 5. Clin Pharmacol Ther. 2021. PMID: 33792051 Free PMC article.
72 results