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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. Among authors: leone d. 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.
Ultrasound assessment of diaphragmatic function in type 1 spinal muscular atrophy.
Buonsenso D, Berti B, Palermo C, Leone D, Ferrantini G, De Sanctis R, Onesimo R, Curatola A, Fanelli L, Forcina N, Norcia G, Carnicella S, Lucibello S, Mercuri E, Pane M. Buonsenso D, et al. Among authors: leone d. Pediatr Pulmonol. 2020 Jul;55(7):1781-1788. doi: 10.1002/ppul.24814. Epub 2020 May 11. Pediatr Pulmonol. 2020. PMID: 32394611
Sometimes they come back: New and old spinal muscular atrophy adults in the era of nusinersen.
Sansone VA, Coratti G, Pera MC, Pane M, Messina S, Salmin F, Albamonte E, De Sanctis R, Sframeli M, Di Bella V, Morando S, d'Amico A, Frongia AL, Antonaci L, Pirola A, Pedemonte M, Bertini E, Bruno C, Mercuri E; Italian ISMAC group. Sansone VA, et al. Eur J Neurol. 2021 Feb;28(2):602-608. doi: 10.1111/ene.14567. Epub 2020 Oct 31. Eur J Neurol. 2021. PMID: 33012052 Free article.
Hypoglycaemia in patients with type 1 SMA: an underdiagnosed problem?
Berti B, Onesimo R, Leone D, Palermo C, Giorgio V, Buonsenso D, Pane M, Mercuri E. Berti B, et al. Among authors: leone d. Arch Dis Child. 2020 Jul;105(7):707. doi: 10.1136/archdischild-2019-318120. Epub 2019 Dec 20. Arch Dis Child. 2020. PMID: 31862697 No abstract available.
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.
Sleep disorders in low-risk preschool very preterm children.
Romeo DM, Leo G, Lapenta L, Leone D, Turrini I, Brogna C, Gallini F, Cota F, Vento G, Mercuri E. Romeo DM, et al. Among authors: leone d. Sleep Med. 2019 Nov;63:137-141. doi: 10.1016/j.sleep.2019.04.016. Epub 2019 May 27. Sleep Med. 2019. PMID: 31627127
254 results