A consortium of European experts has reviewed, four years apart, the use of AAV-based gene therapy in SMA:
- real-life data and feedback from neonatal diagnosis of SMA in many countries were taken into account in this update,
- 19 specialists from 17 European countries, including France, took part in the working group
- twelve proposals were discussed and approved, mostly unanimously,
- these included the possibility of extending the indications to older and/or overweight children, again with the usual precautions.
However, the document does not take a position on cases of SMA detected at birth carrying four copies of the SMN2 gene.
