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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: majumdar a. 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: majumdar a. J Neuromuscul Dis. 2024;11(2):361-368. doi: 10.3233/JND-230162. J Neuromuscul Dis. 2024. PMID: 38189761 Free PMC article.
Therapeutic Role of Nusinersen on Respiratory Progression in Pediatric Patients With Spinal Muscular Atrophy Type 2 and Nonambulant Type 3.
Trucco F, Ridout D, Weststrate H, Scoto M, Rohwer A, Coratti G, Main ML, Mayhew AG, Montes J, De Sanctis R, Pane M, Pera MC, Sansone VA, Albamonte E, D'Amico A, Bruno C, Messina SS, Childs AM, Willis T, Ong MT, Servais L, Majumdar A, Hughes I, Marini-Bettolo C, Parasuraman D, Gowda VL, Baranello G, Bertini ES, De Vivo DC, Darras BT, Day JW, Mayer O, Zolkipli-Cunningham Z, Finkel RS, Mercuri E, Muntoni F; for iSMAc. Trucco F, et al. Among authors: majumdar a. Neurol Clin Pract. 2024 Jun;14(3):e200298. doi: 10.1212/CPJ.0000000000200298. Epub 2024 May 17. Neurol Clin Pract. 2024. PMID: 38932995
Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial.
Mah JK, Clemens PR, Guglieri M, Smith EC, Finkel RS, Tulinius M, Nevo Y, Ryan MM, Webster R, Castro D, Kuntz NL, McDonald CM, Damsker JM, Schwartz BD, Mengle-Gaw LJ, Jackowski S, Stimpson G, Ridout DA, Ayyar-Gupta V, Baranello G, Manzur AY, Muntoni F, Gordish-Dressman H, Leinonen M, Ward LM, Hoffman EP, Dang UJ; NorthStar UK Network and CINRG DNHS Investigators. Mah JK, et al. JAMA Netw Open. 2022 Jan 4;5(1):e2144178. doi: 10.1001/jamanetworkopen.2021.44178. JAMA Netw Open. 2022. PMID: 35076703 Free PMC article. Clinical Trial.
Genotype-phenotype correlation in a large population of muscular dystrophy patients with LAMA2 mutations.
Geranmayeh F, Clement E, Feng LH, Sewry C, Pagan J, Mein R, Abbs S, Brueton L, Childs AM, Jungbluth H, De Goede CG, Lynch B, Lin JP, Chow G, Sousa Cd, O'Mahony O, Majumdar A, Straub V, Bushby K, Muntoni F. Geranmayeh F, et al. Among authors: majumdar a. Neuromuscul Disord. 2010 Apr;20(4):241-50. doi: 10.1016/j.nmd.2010.02.001. Epub 2010 Mar 6. Neuromuscul Disord. 2010. PMID: 20207543
Growth pattern trajectories in boys with Duchenne muscular dystrophy.
Stimpson G, Raquq S, Chesshyre M, Fewtrell M, Ridout D, Sarkozy A, Manzur A, Ayyar Gupta V, De Amicis R, Muntoni F, Baranello G; NorthStar Network. Stimpson G, et al. Orphanet J Rare Dis. 2022 Jan 24;17(1):20. doi: 10.1186/s13023-021-02158-9. Orphanet J Rare Dis. 2022. PMID: 35073949 Free PMC article.
1,598 results