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Gene therapy as a potential therapeutic option for Duchenne muscular dystrophy: A qualitative preference study of patients and parents.
Landrum Peay H, Fischer R, Tzeng JP, Hesterlee SE, Morris C, Strong Martin A, Rensch C, Smith E, Ricotti V, Beaverson K, Wand H, Mansfield C. Landrum Peay H, et al. Among authors: ricotti v. PLoS One. 2019 May 1;14(5):e0213649. doi: 10.1371/journal.pone.0213649. eCollection 2019. PLoS One. 2019. PMID: 31042754 Free PMC article.
Safety, Tolerability, and Pharmacokinetics of SMT C1100, a 2-Arylbenzoxazole Utrophin Modulator, following Single- and Multiple-Dose Administration to Pediatric Patients with Duchenne Muscular Dystrophy.
Ricotti V, Spinty S, Roper H, Hughes I, Tejura B, Robinson N, Layton G, Davies K, Muntoni F, Tinsley J. Ricotti V, et al. PLoS One. 2016 Apr 7;11(4):e0152840. doi: 10.1371/journal.pone.0152840. eCollection 2016. PLoS One. 2016. PMID: 27055247 Free PMC article. Clinical Trial.
Revised North Star Ambulatory Assessment for Young Boys with Duchenne Muscular Dystrophy.
Mercuri E, Coratti G, Messina S, Ricotti V, Baranello G, D'Amico A, Pera MC, Albamonte E, Sivo S, Mazzone ES, Arnoldi MT, Fanelli L, De Sanctis R, Romeo DM, Vita GL, Battini R, Bertini E, Muntoni F, Pane M. Mercuri E, et al. Among authors: ricotti v. PLoS One. 2016 Aug 5;11(8):e0160195. doi: 10.1371/journal.pone.0160195. eCollection 2016. PLoS One. 2016. PMID: 27494024 Free PMC article.
Upper Limb Evaluation in Duchenne Muscular Dystrophy: Fat-Water Quantification by MRI, Muscle Force and Function Define Endpoints for Clinical Trials.
Ricotti V, Evans MR, Sinclair CD, Butler JW, Ridout DA, Hogrel JY, Emira A, Morrow JM, Reilly MM, Hanna MG, Janiczek RL, Matthews PM, Yousry TA, Muntoni F, Thornton JS. Ricotti V, et al. PLoS One. 2016 Sep 20;11(9):e0162542. doi: 10.1371/journal.pone.0162542. eCollection 2016. PLoS One. 2016. PMID: 27649492 Free PMC article.
Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best-worst scaling experiment in caregivers and adult patients.
Paquin RS, Fischer R, Mansfield C, Mange B, Beaverson K, Ganot A, Martin AS, Morris C, Rensch C, Ricotti V, Russo LJ, Sadosky A, Smith EC, Peay HL. Paquin RS, et al. Among authors: ricotti v. Orphanet J Rare Dis. 2019 May 9;14(1):102. doi: 10.1186/s13023-019-1069-6. Orphanet J Rare Dis. 2019. PMID: 31072340 Free PMC article.
Patients' and caregivers' maximum acceptable risk of death for non-curative gene therapy to treat Duchenne muscular dystrophy.
Peay HL, Fischer R, Mange B, Paquin RS, Smith EC, Sadosky A, Russo L, Ricotti V, Rensch C, Morris C, Martin AS, Ganot A, Beaverson K, Mansfield C. Peay HL, et al. Among authors: ricotti v. Mol Genet Genomic Med. 2021 May;9(5):e1664. doi: 10.1002/mgg3.1664. Epub 2021 Mar 23. Mol Genet Genomic Med. 2021. PMID: 33755338 Free PMC article.
Early neurodevelopmental assessment in Duchenne muscular dystrophy.
Pane M, Scalise R, Berardinelli A, D'Angelo G, Ricotti V, Alfieri P, Moroni I, Hartley L, Pera MC, Baranello G, Catteruccia M, Casalino T, Romeo DM, Graziano A, Gandioli C, Bianco F, Mazzone ES, Lombardo ME, Scoto M, Sivo S, Palermo C, Gualandi F, Sormani MP, Ferlini A, Bertini E, Muntoni F, Mercuri E. Pane M, et al. Among authors: ricotti v. Neuromuscul Disord. 2013 Jun;23(6):451-5. doi: 10.1016/j.nmd.2013.02.012. Epub 2013 Mar 25. Neuromuscul Disord. 2013. PMID: 23535446
90 results