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.
- 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.