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448 results

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'Fighting every day': exploring caregiver quality of life and perspectives on healthcare services for children with dementia - a cross-sectional, mixed-methods study.
Djafar J, Nevin S, Smith N, Ardern-Holmes S, Bhattacharya K, Dale R, Ellaway C, Grattan S, Johnson A, Kandula T, Kariyawasam DS, Lewis K, Meagher CE, Mohammad S, Farrar MA. Djafar J, et al. Among authors: farrar ma. Arch Dis Child. 2025 Jan 19:archdischild-2024-328011. doi: 10.1136/archdischild-2024-328011. Online ahead of print. Arch Dis Child. 2025. PMID: 39832815
Safety and Efficacy of IV Onasemnogene Abeparvovec for Pediatric Patients With Spinal Muscular Atrophy: The Phase 3b SMART Study.
McMillan HJ, Baranello G, Farrar MA, Zaidman CM, Moreno T, De Waele L, Jong YJ, Laugel V, Quijano-Roy S, Mercuri E, Chien YH, Straub V, Darras BT, Seibert J, Bernardo Escudero R, Alecu I, Freischläger F, Muntoni F; SMART Study Group. McMillan HJ, et al. Among authors: farrar ma. Neurology. 2025 Jan 28;104(2):e210268. doi: 10.1212/WNL.0000000000210268. Epub 2024 Dec 30. Neurology. 2025. PMID: 39804575 Free PMC article. Clinical Trial.
Nationwide, Couple-Based Genetic Carrier Screening.
Kirk EP, Delatycki MB, Archibald AD, Tutty E, Caruana J, Halliday JL, Lewis S, McClaren BJ, Newson AJ, Dive L, Best S, Long JC, Braithwaite J, Downes MJ, Scuffham PA, Massie J, Barlow-Stewart K, Kulkarni A, Ruscigno A, Kanga-Parabia A, Rodrigues B, Bennetts BH, Ebzery C, Hunt C, Cliffe CC, Lee C, Azmanov D, King EA, Madelli EO, Zhang F, Ho G, Danos I, Liebelt J, Fletcher J, Kennedy J, Beilby J, Emery JD, McGaughran J, Marum JE, Scarff K, Fisk K, Harrison K, Boggs K, Giameos L, Fitzgerald L, Thomas L, Burnett L, Freeman L, Harris M, Berbic M, Davis MR, Cifuentes Ochoa M, Wallis M, Wall M, Chow MTM, Ferrie MM, Pachter N, Quayum N, Lang N, Kasi Pandy P, Casella R, Allcock RJN, Ong R, Edwards S, Sundercombe S, Jelenich S, Righetti S, Lunke S, Kaur S, Stock-Myer S, Eggers S, Walker SP, Theodorou T, Catchpool T, Clinch T, Roscioli T, Hardy T, Zhu Y, Fehlberg Z, Boughtwood TF, Laing NG; Mackenzie’s Mission Investigators; Mackenzie's Mission Investigators. Kirk EP, et al. N Engl J Med. 2024 Nov 21;391(20):1877-1889. doi: 10.1056/NEJMoa2314768. N Engl J Med. 2024. PMID: 39565987
A contemporary analysis of the Australian clinical and genetic landscape of spinal muscular atrophy: a registry based study.
Balaji L, Forbes R, Cairns A, Sampaio H, Kornberg AJ, Sanders L, Lamont P, Liang C, Jones KJ, Nowak K, O'Gorman C, Woodcock I, Briggs N, Yiu EM, Farrar MA, Kariyawasam D. Balaji L, et al. Among authors: farrar ma. Lancet Reg Health West Pac. 2024 Nov 6;53:101237. doi: 10.1016/j.lanwpc.2024.101237. eCollection 2024 Dec. Lancet Reg Health West Pac. 2024. PMID: 39559164 Free PMC article.
Codesign and evaluation of advanced therapeutic information resources for and with families of children with neurological conditions: a mixed methods cross-sectional study.
Meagher CE, Kariyawasam DS, Concepcion KAE, Dale R, Hetherington K, Mohammad S, Palmer EE, Woolfenden S, Farrar MA. Meagher CE, et al. Among authors: farrar ma. Arch Dis Child. 2024 Nov 19:archdischild-2024-327914. doi: 10.1136/archdischild-2024-327914. Online ahead of print. Arch Dis Child. 2024. PMID: 39521449
Outcomes of early-treated infants with spinal muscular atrophy: A multicenter, retrospective cohort study.
Goedeker NL, Rogers A, Fisher M, Arya K, Brandsema JF, Farah H, Farrar MA, Felker MV, Gibbons M, Hamid OA, Harmelink M, Herbert K, Kichula E, King K, Lakhotia A, Lee BH, Kuntz NL, Parsons J, Rehborg R, Veerapaniyan A, Zaidman CM. Goedeker NL, et al. Among authors: farrar ma. Muscle Nerve. 2024 Dec;70(6):1247-1256. doi: 10.1002/mus.28267. Epub 2024 Oct 6. Muscle Nerve. 2024. PMID: 39370660
A feasibility study of controlled human infection with intradermal Bacillus Calmette-Guérin (BCG) injection: Pilot BCG controlled human infection model.
Carter E, Morton B, ElSafadi D, Jambo K, Kenny-Nyazika T, Hyder-Wright A, Chiwala G, Chikaonda T, Chirwa AE, Gonzalez Sanchez J, Yip V, Biagini G, Pennington SH, Saunderson P, Farrar M, Myerscough C, Collins AM, Gordon SB, Ferreira DM. Carter E, et al. Among authors: farrar m. Wellcome Open Res. 2024 Jun 5;8:424. doi: 10.12688/wellcomeopenres.19811.2. eCollection 2023. Wellcome Open Res. 2024. PMID: 39219857 Free PMC article.
448 results