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 risdiplam
• Not have had successful treatment with Zolgensma.

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

If you or your child meet the eligibility criteria, you must also meet these starting 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 do not meet this individual criterion.

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

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

Other exclusions are if you, your family or your carer:

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

The broader Standards of Care (SoC) for SMA can be  found in the Family Guide to the SoC.  These standards are currently under review for the UK. See SMA Care UK >.

We answer: Can children who have had Zolgensma have risdiplam? > 

The NICE Website has a Question and Answer page > This covers nusinersen and risdiplam.