Real-world Adherence to Nusinersen in Adults with Spinal Muscular Atrophy in the US: A Multi-site Chart Review Study.
| Autor: | Elman L; University of Pennsylvania, Philadelphia, PA, USA., Youn B; Biogen, Cambridge, MA, USA., Proud CM; Children's Hospital of the King's Daughters, Norfolk, VA, USA., Frey MR; Memorial Healthcare Institute for Neuroscience, Owosso, MI, USA., Ajroud-Driss S; Northwestern University Feinberg School of Medicine, Chicago, IL, USA., McCormick ME; Oakland University William Beaumont School of Medicine, Royal Oak, MI, USA., Michelson D; Loma Linda University Health, Loma Linda, CA, USA., Cartwright MS; Wake Forest School of Medicine, Winston-Salem, NC, USA., Heiman-Patterson T; Lewis Katz School of Medicine, Temple University, Philadelphia, PA, USA., Choi JM; Georgetown University Hospital, Washington, DC, USA., Chandak A; Certara, New York, NY, USA., Khachatryan A; Certara, London, UK., Martinez M; Certara, London, UK., Paradis AD; Biogen, Cambridge, MA, USA. |
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| Jazyk: | angličtina |
| Zdroj: | Journal of neuromuscular diseases [J Neuromuscul Dis] 2022; Vol. 9 (5), pp. 655-660. |
| DOI: | 10.3233/JND-210768 |
| Abstrakt: | Limited evidence exists on real-world adherence to nusinersen for the treatment of spinal muscular atrophy (SMA). Data are presented from a multi-site retrospective chart review of 86 adults with SMA initiating nusinersen at nine US centers between January 2017 and February 2019. Seventy-nine (92%) adults remained on nusinersen during the study; 454 (92%) of 493 total nusinersen doses were received on time. Fifty-eight (67%) adults received all nusinersen doses on time. The majority of patients with at least one nonadherent dose resumed nusinersen on time. Most patients followed the dosing schedule across the loading and maintenance dose periods. |
| Databáze: | MEDLINE |
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