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Page 1
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. 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.
Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls.
Darras BT, Masson R, Mazurkiewicz-Bełdzińska M, Rose K, Xiong H, Zanoteli E, Baranello G, Bruno C, Vlodavets D, Wang Y, El-Khairi M, Gerber M, Gorni K, Khwaja O, Kletzl H, Scalco RS, Fontoura P, Servais L; FIREFISH Working Group. Darras BT, et al. N Engl J Med. 2021 Jul 29;385(5):427-435. doi: 10.1056/NEJMoa2102047. N Engl J Med. 2021. PMID: 34320287
Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial.
Masson R, Mazurkiewicz-Bełdzińska M, Rose K, Servais L, Xiong H, Zanoteli E, Baranello G, Bruno C, Day JW, Deconinck N, Klein A, Mercuri E, Vlodavets D, Wang Y, Dodman A, El-Khairi M, Gorni K, Jaber B, Kletzl H, Gaki E, Fontoura P, Darras BT; FIREFISH Study Group. Masson R, et al. Lancet Neurol. 2022 Dec;21(12):1110-1119. doi: 10.1016/S1474-4422(22)00339-8. Epub 2022 Oct 14. Lancet Neurol. 2022. PMID: 36244364 Clinical Trial.
Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial.
Mercuri E, Deconinck N, Mazzone ES, Nascimento A, Oskoui M, Saito K, Vuillerot C, Baranello G, Boespflug-Tanguy O, Goemans N, Kirschner J, Kostera-Pruszczyk A, Servais L, Gerber M, Gorni K, Khwaja O, Kletzl H, Scalco RS, Staunton H, Yeung WY, Martin C, Fontoura P, Day JW; SUNFISH Study Group. Mercuri E, et al. Lancet Neurol. 2022 Jan;21(1):42-52. doi: 10.1016/S1474-4422(21)00367-7. Lancet Neurol. 2022. PMID: 34942136
The Complement System in Kidney Transplantation.
Santarsiero D, Aiello S. Santarsiero D, et al. Cells. 2023 Mar 2;12(5):791. doi: 10.3390/cells12050791. Cells. 2023. PMID: 36899927 Free PMC article. Review.
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: santarsiero d. 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.
Exuberant Endothelial C5b-9 Formation in Recurrent and De Novo Posttransplant Thrombotic Microangiopathy.
Aiello S, Gastoldi S, Bresin E, Galbusera M, Mele C, Daina E, Santarsiero D, Comai G, La Manna G, Martinatto C, Benigni A, Remuzzi G, Noris M. Aiello S, et al. Among authors: santarsiero d. Kidney Int Rep. 2024 Aug 22;9(11):3318-3323. doi: 10.1016/j.ekir.2024.08.014. eCollection 2024 Nov. Kidney Int Rep. 2024. PMID: 39534196 Free PMC article. No abstract available.
Retinal Photoreceptor Functions Are Compromised in Patients With Resistance to Thyroid Hormone Syndrome (RTHβ).
Campi I, Cammarata G, Bianchi Marzoli S, Beck-Peccoz P, Santarsiero D, Dazzi D, Bottari de Castello A, Taroni EG, Viola F, Mian C, Watutantrige-Fernando S, Pelusi C, Muzza M, Maffini MA, Persani L. Campi I, et al. Among authors: santarsiero d. J Clin Endocrinol Metab. 2017 Jul 1;102(7):2620-2627. doi: 10.1210/jc.2016-3671. J Clin Endocrinol Metab. 2017. PMID: 28379567