Landfeldt, Erik;
Udo, Camilla;
Cortina-Borja, Mario;
Sejersen, Thomas;
Kreicbergs, Ulrika;
(2025)
“Not Only Has She Survived, But She Lives a Happy Life”: Parents’ Perspectives and Experiences of a Novel Disease-Modifying Therapy for Spinal Muscular Atrophy in Sweden.
Journal of Child Neurology
10.1177/08830738251346921.
(In press).
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Abstract
The objective of this prospective cohort study was to explore parents' perspectives of patients' experiences of the first available novel disease-modifying therapy for SMA in Sweden. Patients with SMA and their parents/legal guardians were identified in the National Patient Register and the Multi-Generation Register. Data was recorded using an electronic questionnaire administered at baseline, and after 6, 12, and 18 months. In total, 47 parents to 33 children with SMA (mean patient age: 9 years, 59% female; 27% with SMA type I, 33% with type II, and 39% with type III) participated. All parents reported that they wished their child to be treated with nusinersen and most parents (81%) reported that they had sufficient information to make an informed treatment decision. Across follow-up, almost all parents reported having a positive experience of nusinersen. Our study provides unique insights into caregivers' real-world experiences of a novel disease-modifying therapy for SMA.
| Type: | Article |
|---|---|
| Title: | “Not Only Has She Survived, But She Lives a Happy Life”: Parents’ Perspectives and Experiences of a Novel Disease-Modifying Therapy for Spinal Muscular Atrophy in Sweden |
| Location: | United States |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1177/08830738251346921 |
| Publisher version: | https://doi.org/10.1177/08830738251346921 |
| Language: | English |
| Additional information: | This version is the author accepted manuscript. For information on re-use, please refer to the publisher's terms and conditions. |
| Keywords: | Clinical Decision-Making; Information Sources; Caregiver Burden; Treatment Outcome; nusinersen |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Population, Policy and Practice Dept |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10212211 |
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