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Table 1 Summary of main start and stop criteria for nusinersen treatment in Norway [13]

From: Priority setting at the clinical level: the case of nusinersen and the Norwegian national expert group

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