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Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial.
Servais L, Mercuri E, Straub V, Guglieri M, Seferian AM, Scoto M, Leone D, Koenig E, Khan N, Dugar A, Wang X, Han B, Wang D, Muntoni F; SKIP-NMD Study Group. Servais L, et al. Nucleic Acid Ther. 2022 Feb;32(1):29-39. doi: 10.1089/nat.2021.0043. Epub 2021 Nov 17. Nucleic Acid Ther. 2022. PMID: 34788571 Free PMC article. Clinical Trial.
Congenital lung malformations: a novel application for lung ultrasound?
Merli L, Nanni L, Curatola A, Pellegrino M, De Santis M, Silvaroli S, Paradiso FV, Buonsenso D. Merli L, et al. J Ultrasound. 2021 Sep;24(3):349-353. doi: 10.1007/s40477-019-00406-0. Epub 2019 Sep 5. J Ultrasound. 2021. PMID: 31487034 Free PMC article. Review.
Safety and efficacy of ruxolitinib in splanchnic vein thrombosis associated with myeloproliferative neoplasms.
Pieri L, Paoli C, Arena U, Marra F, Mori F, Zucchini M, Colagrande S, Castellani A, Masciulli A, Rosti V, De Stefano V, Betti S, Finazzi G, Ferrari ML, Rumi E, Ruggeri M, Nichele I, Guglielmelli P, Fjerza R, Mannarelli C, Fanelli T, Merli L, Corbizi Fattori G, Massa M, Cimino G, Rambaldi A, Barosi G, Cazzola M, Barbui T, Vannucchi AM. Pieri L, et al. Among authors: merli l. Am J Hematol. 2017 Feb;92(2):187-195. doi: 10.1002/ajh.24614. Am J Hematol. 2017. PMID: 27880982 Free article. Clinical Trial.
20 results