You or your child must:

• Have had a confirmed diagnosis of 5q autosomal recessive SMA – an SMN1 gene deletion test and
• A clinical diagnosis of SMA Type 1, Type 2 or Type 3

or

• Have pre-symptomatic SMA with one to four SMN2 copies

Also, you or your child must:

• Not be receiving another treatment i.e., it cannot be combined with nusinersen
• Not have had successful treatment with onasemnogene abeparvovec. The definition of this can be found here.

There is no upper age limit for when treatment may start.

If you / your child meets the eligibility criteria, you also need to meet these starting / entry criteria.

You or your child must:

• Not be on permanent ventilation. This is when you / your child needs to use a non-invasive ventilator (NIV such as BiPaP) for more than 16 hours/day for 21 consecutive days and this is not because of an acute reversible chest infection. Patients who do not meet this criterion but otherwise meet the eligibility criteria can be referred for advice to the NHS England SMA Clinical Panel.
• Not have a tracheostomy. Again, patients who do not meet this criterion but otherwise meet the eligibility criteria can be referred for advice to the NHS England SMA Clinical Panel. Access may be potentially possible, even if you / your child doesn’t meet this individual criterion.

These describe any conditions that may prevent you / your child from receiving treatment within the MAA. These include if a child, young person or adult is diagnosed with an additional progressive life limiting condition where treatment with risdiplam would not provide long- term benefit, such as terminal cancer or catastrophic brain injury.

Other exclusions are if you / your family / your carer:

• are unwilling to comply with required monitoring criteria that are explained to them
• refuses to sign the informed consent form – the Managed Access Patient Agreement
• is unwilling to cooperate with the treatment centre so that you /your child receive the appropriate standard of care management for your/your child’s condition and circumstances, especially for nutrition and respiratory
  care and vaccinations, such as influenza and
  respiratory syncytial virus (RSV).

The broader internationally recommended Standards of Care (SoC) for SMA can be found in the Family Guide to the SoC.

We asked NHS England this question and on 11th March 2022, they advised that:

"…clinicians should prioritise individuals who have had recent deterioration in walking, feeding or ventilatory requirements. It would also be important to take into account when individuals were already scheduled to see their clinician to plan future treatment and not to disrupt these appointments."