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Page 1
Long-term Natural History of Pediatric Dominant and Recessive RYR1-Related Myopathy.
Sarkozy A, Sa M, Ridout D, Fernandez-Garcia MA, Distefano MG, Main M, Sheehan J, Manzur AY, Munot P, Robb S, Wraige E, Quinlivan R, Scoto M, Baranello G, Gowda V, Mein R, Phadke R, Jungbluth H, Muntoni F. Sarkozy A, et al. Among authors: baranello g. Neurology. 2023 Oct 10;101(15):e1495-e1508. doi: 10.1212/WNL.0000000000207723. Epub 2023 Aug 29. Neurology. 2023. PMID: 37643885 Free PMC article.
Muscle magnetic resonance imaging involvement patterns in nemaline myopathies.
Perry L, Stimpson G, Singh L, Morrow JM, Shah S, Baranello G, Muntoni F, Sarkozy A. Perry L, et al. Among authors: baranello g. Ann Clin Transl Neurol. 2023 Jul;10(7):1219-1229. doi: 10.1002/acn3.51816. Epub 2023 Jun 2. Ann Clin Transl Neurol. 2023. PMID: 37265148 Free PMC article.
Quantifying Variability in Motor Function in Duchenne Muscular Dystrophy: UK Centiles for the NorthStar Ambulatory Assessment, 10 m Walk Run Velocity and Rise from Floor Velocity in GC Treated Boys.
Stimpson G, Ridout D, Wolfe A, Milev E, O'Reilly E, Manzur A, Sarkozy A, Muntoni F, Cole TJ, Baranello G; NorthStar Network. Stimpson G, et al. Among authors: baranello g. J Neuromuscul Dis. 2024;11(1):153-166. doi: 10.3233/JND-230159. J Neuromuscul Dis. 2024. PMID: 37980680 Free PMC article.
Efficacy and safety of onasemnogene abeparvovec in children with spinal muscular atrophy type 1: real-world evidence from 6 infusion centres in the United Kingdom.
Gowda V, Atherton M, Murugan A, Servais L, Sheehan J, Standing E, Manzur A, Scoto M, Baranello G, Munot P, McCullagh G, Willis T, Tirupathi S, Horrocks I, Dhawan A, Eyre M, Vanegas M, Fernandez-Garcia MA, Wolfe A, Pinches L, Illingworth M, Main M, Abbott L, Smith H, Milton E, D'Urso S, Vijayakumar K, Marco SS, Warner S, Reading E, Douglas I, Muntoni F, Ong M, Majumdar A, Hughes I, Jungbluth H, Wraige E. Gowda V, et al. Among authors: baranello g. Lancet Reg Health Eur. 2023 Dec 11;37:100817. doi: 10.1016/j.lanepe.2023.100817. eCollection 2024 Feb. Lancet Reg Health Eur. 2023. PMID: 38169987 Free PMC article.
Risdiplam in Spinal Muscular Atrophy: Safety Profile and Use Through The Early Access to Medicine Scheme for the Paediatric Cohort in Great Britain.
Cornell N, Childs AM, Wraige E, Munot P, Ambegaonkar G, Chow G, Hughes I, Illingworth M, Majumdar A, Marini-Bettolo C, Parasuraman D, Spinty S, Willis T, Scoto M, Baranello G; Paediatric UK Risdiplam EAMS Working Group. Cornell N, et al. Among authors: baranello g. J Neuromuscul Dis. 2024;11(2):361-368. doi: 10.3233/JND-230162. J Neuromuscul Dis. 2024. PMID: 38189761 Free PMC article.
Secondary outcomes of scoliosis surgery in disease-modifying treatment-naïve patients with spinal muscular atrophy type 2 and nonambulant type 3.
Brusa C, Baranello G, Ridout D, de Graaf J, Manzur AY, Munot P, Sarkozy A, Main M, Milev E, Iodice M, Ramsey D, Tucker S, Ember T, Nadarajah R, Muntoni F, Scoto M. Brusa C, et al. Among authors: baranello g. Muscle Nerve. 2024 Nov;70(5):1000-1009. doi: 10.1002/mus.28238. Epub 2024 Sep 4. Muscle Nerve. 2024. PMID: 39233378
172 results