SMA Europe responds to European neuromuscular experts’ consensus statements on gene replacement therapy for SMA
A group of European neuromuscular experts presented eleven consensus statements covering qualification, patient selection, safety considerations and long-term monitoring, in order to aid the rational use of Zolgensma™ for the treatment of SMA (1).
In a letter published in the European Journal of Paediatric Neurology (2), SMA Europe addresses these consensus statements by highlights:
- The challenging decisions families with a child with SMA face in the light of several treatments now being available
- That in order to make informed decisions, families need to understand the full picture of the disease, including the patient perspective on quality of life.
- That it is essential to have a transparent discussion about the range of potential outcomes of different treatments, followed by shared decision-making.
- That predictive factors for treatment outcomes remain unclear, therefore potential treatment benefit must be assessed on an individual level.
Full access to our position paper is available for 50 days.
References
- Kirschner, J., Butoianu, N., Goemans, N., Haberlova, J., Kostera-Pruszczyk, A., Mercuri, E., . . . Muntoni, F. (2020, September 01). European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy. European Journal of Paediatric Neurology, 28, 38 - 43.
- Gusset, N., Erbas, Y., Germanenko, O., Rucinski, K., Stumpe, E., & de Lemus, M. (2020). A Decision for Life – Treatment decisions in newly diagnosed families with spinal muscular atrophy (SMA). European Journal of Paediatric Neurology. In press.