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Year Number of Results
1973 1
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Page 1
Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial.
Mercuri E, Muntoni F, Baranello G, Masson R, Boespflug-Tanguy O, Bruno C, Corti S, Daron A, Deconinck N, Servais L, Straub V, Ouyang H, Chand D, Tauscher-Wisniewski S, Mendonca N, Lavrov A; STR1VE-EU study group. Mercuri E, et al. Lancet Neurol. 2021 Oct;20(10):832-841. doi: 10.1016/S1474-4422(21)00251-9. Lancet Neurol. 2021. PMID: 34536405 Clinical Trial.
Risdiplam in Presymptomatic Spinal Muscular Atrophy.
Finkel RS, Servais L, Vlodavets D, Zanoteli E, Mazurkiewicz-Bełdzińska M, Jong YJ, Navas-Nazario A, Al-Muhaizea M, Araujo APQC, Nelson L, Wang Y, Jaber B, Gorni K, Kletzl H, Palfreeman L, Rabbia M, Summers D, Gaki E, Wagner KR, Fontoura P, Farrar MA, Bertini E; RAINBOWFISH Study Group. Finkel RS, et al. N Engl J Med. 2025 Aug 14;393(7):671-682. doi: 10.1056/NEJMoa2410120. N Engl J Med. 2025. PMID: 40802943 Clinical Trial.
Searching the undetected mtDNA variants in forensic MPS data.
Melchionda F, Stanciu F, Buscemi L, Pesaresi M, Tagliabracci A, Turchi C. Melchionda F, et al. Among authors: buscemi l. Forensic Sci Int Genet. 2020 Nov;49:102399. doi: 10.1016/j.fsigen.2020.102399. Epub 2020 Sep 28. Forensic Sci Int Genet. 2020. PMID: 33038616
70 results