Start criteria |
Minimum two copies of the SMN2 gene |
Patient should not be dependent on assisted ventilation or extra oxygen to keep an SaO2 > 95% |
Regarding SMA 3, patients with onset before three years of age may in certain situations be eligible for treatment |
Stop criteria |
SMA 1: Reduction in score on defined motor tests or deterioration regarding nutritional and respiratory status despite treatment |
In need of assisted ventilation more than 16 h/day |
SMA 2 and 3: Reduction in score on defined motor tests. Deterioration in respiratory status |