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Page 1
Assessing Bulbar Function in Spinal Muscular Atrophy Using Patient-Reported Outcomes.
Dunaway Young S, Pasternak A, Duong T, McGrattan KE, Stranberg S, Maczek E, Dias C, Tang W, Parker D, Levine A, Rohan A, Wolford C, Martens W, McDermott MP, Darras BT, Day JW. Dunaway Young S, et al. Among authors: levine a. J Neuromuscul Dis. 2023;10(2):199-209. doi: 10.3233/JND-221573. J Neuromuscul Dis. 2023. PMID: 36776075 Free PMC article.
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
Natural history of Type 1 spinal muscular atrophy: a retrospective, global, multicenter study.
Cances C, Vlodavets D, Comi GP, Masson R, Mazurkiewicz-Bełdzińska M, Saito K, Zanoteli E, Dodman A, El-Khairi M, Gorni K, Gravestock I, Hoffart J, Scalco RS, Darras BT; ANCHOVY Working Group. Cances C, et al. Orphanet J Rare Dis. 2022 Jul 29;17(1):300. doi: 10.1186/s13023-022-02455-x. Orphanet J Rare Dis. 2022. PMID: 35906608 Free PMC article.
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.
Early trajectories of virological and immunological biomarkers and clinical outcomes in patients admitted to hospital for COVID-19: an international, prospective cohort study.
Jensen TO, Murray TA, Grandits GA, Jain MK, Grund B, Shaw-Saliba K, Matthay MA, Abassi M, Ardelt M, Baker JV, Chen P, Dewar RL, Goodman AL, Hatlen TJ, Highbarger HC, Holodniy M, Lallemand P, Laverdure S, Leshnower BG, Looney D, Moschopoulos CD, Mugerwa H, Murray DD, Mylonakis E, Nagy-Agren S, Rehman MT, Rupert A, Stevens R, Turville S, Weintrob A, Wick K, Lundgren J, Ko ER; ACTIV-3/TICO Study Group. Jensen TO, et al. Lancet Microbe. 2024 May 27:S2666-5247(24)00015-6. doi: 10.1016/S2666-5247(24)00015-6. Online ahead of print. Lancet Microbe. 2024. PMID: 38815595 Free article.
A contact binary satellite of the asteroid (152830) Dinkinesh.
Levison HF, Marchi S, Noll KS, Spencer JR, Statler TS, Bell JF 3rd, Bierhaus EB, Binzel R, Bottke WF, Britt D, Brown ME, Buie MW, Christensen PR, Dello Russo N, Emery JP, Grundy WM, Hahn M, Hamilton VE, Howett C, Kaplan H, Kretke K, Lauer TR, Manzoni C, Marschall R, Martin AC, May BH, Mottola S, Olkin CB, Pätzold M, Parker JW, Porter S, Preusker F, Protopapa S, Reuter DC, Robbins SJ, Salmon J, Simon AA, Stern SA, Sunshine JM, Wong I, Weaver HA, Adam C, Ancheta S, Andrews J, Anwar S, Barnouin OS, Beasley M, Berry KE, Birath E, Bolin B, Booco M, Burns R, Campbell P, Carpenter R, Crombie K, Effertz M, Eifert E, Ellis C, Faiks P, Fischetti J, Fleming P, Francis K, Franco R, Freund S, Gallagher C, Geeraert J, Gobat C, Gorgas D, Granat C, Gray S, Haas P, Harch A, Hegedus K, Isabelle C, Jackson B, Jacob T, Jennings S, Kaufmann D, Keeney BA, Kennedy T, Lauffer K, Lessac-Chenen E, Leonard R, Levine A, Lunsford A, Martin T, McAdams J, Mehall G, Merkley T, Miller G, Montanaro M, Montgomery A, Murphy G, Myers M, Nelson DS, Ocampo A, Olds R, Pelgrift JY, Perkins T, Pineau J, Poland D, Ramanan V, Rose D, Sahr E, Short O, Solanki I, Stanbridge D, Sutter B, Talpas Z, Taylor H, Treiu B, Ver… See abstract for full author list ➔ Levison HF, et al. Among authors: levine a. Nature. 2024 May;629(8014):1015-1020. doi: 10.1038/s41586-024-07378-0. Epub 2024 May 29. Nature. 2024. PMID: 38811709 Free PMC article.
Understanding diffuse leptomeningeal glioneuronal tumors.
Bajin IY, Levine A, Dewan MC, Bennett J, Tabori U, Hawkins C, Bouffet E. Bajin IY, et al. Among authors: levine a. Childs Nerv Syst. 2024 May 25. doi: 10.1007/s00381-024-06432-6. Online ahead of print. Childs Nerv Syst. 2024. PMID: 38795167
3,583 results