- •With several treatments available, families with a child with SMA are faced with challenging decisions.
- •In order to make informed decisions, families need to understand the full picture of the disease, including the patient perspective on quality of life.
- •A transparent discussion about the range of potential outcomes of different treatments, followed by shared decision-making is essential.
- •Predictive factors for treatment outcomes remain unclear, therefore potential treatment benefit must be assessed on an individual level.
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- European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy.Eur. J. Paediatr. Neurol. 2020; https://doi.org/10.1016/j.ejpn.2020.07.001
- Gene therapy for spinal muscular atrophy: Solomon's consensus in Covid times.Eur. J. Paediatr. Neurol. 2020; https://doi.org/10.1016/j.ejpn.2020.08.005
- Self-reported quality of life has no correlation with functional status in children and adolescents with spinal muscular atrophy.Eur. J. Paediatr. Neurol. 2011; 15: 36-39https://doi.org/10.1016/j.ejpn.2010.07.003
N. Gusset, et al., Understanding European Patient Expectations towards Current Therapeutic Development in Spinal Muscular Atrophy (submitted).
Published online: December 14, 2020
Accepted: November 20, 2020
Received: September 28, 2020
© 2020 European Paediatric Neurology Society. Published by Elsevier Ltd. All rights reserved.
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- Response to letter: A decision for life – Treatment decisions in newly diagnosed families with spinal muscular atrophyEuropean Journal of Paediatric NeurologyVol. 30
- Previewwe highly appreciate the comment by Gusset et al.  on our ad-hoc consensus statement , as input from patient organisations is instrumental to ensure that patients and their families remain in the focus of the debate. We fully support the demand of the authors for data-driven education, transparent communication to families and patients, and a balanced public discussion in the context of new available treatments for spinal muscular atrophy ( SMA ).