You should discuss the options with your treatment centre. If your local treating centre needs advice, the MAA Clinical Panel can offer the clinical team advice on the feasibility of administration of the drug following surgery.

You should discuss your child’s ventilation requirements and the reasons for this and your wish for your child to have access to treatment with your treatment centre. Do not change your child’s treatment or ventilation plan without prior discussion and agreement with your treating clinician.

Treatment centres will need to carefully schedule treatments, especially the loading doses. If your child is unwell, it may mean that they will not be able to start treatment on the expected date, but they should still be prioritised in line with any NHS England priority guidelines.

Recovery time is different for each patient. If it is the first time your child has been given the treatment, or the procedure is complicated, or your child needs sedation, you can expect they may need longer in hospital. If the procedure is very straight forward and they have received it multiple times previously then a minimal time in hospital may be all that your child needs. You can talk this through with your medical team.

Individual treatment centres will have staff trained in administering intrathecal injections. They will be able to explain what the treatment involves and whether, for example, sedation or anaesthesia are appropriate.

You should ask your treatment centre about any possible accommodation options.

If you’re in receipt of benefits and/or on a low income, you can get help with travel costs through the Healthcare Costs Travel Scheme. You should ask your treatment centre about any possible accommodation options. You can also ask your clinical team – in particular the Neuromuscular Care Advisor or Nurse Liaison – or SMA UK’s Support & Outreach Team if there are any other possible sources of financial help.

Your child should have their treatment as soon as possible after the one they have missed. You should discuss this with the treatment centre. There should always be at least 14 days between treatments.

The time it takes to see a response from therapy is variable between patients and depends on many different factors. Some patients respond very quickly, within months, others take longer, up to a year. Families with children have sometimes reported responses before they appear on motor assessment scores. A rule of thumb is that the more unwell the patient or the longer the patient has lived with SMA, the longer it may take to show benefit from therapy.

See Clinical Assessment Scales and Measures – what’s used, when?

Many hospitals can provide emotional support services. If this service does not exist at your hospital, you should consider contacting your GP for local emotional support services. The SMA patient charities are also a good source of support.

It is expected that individuals will transition from paediatric to adult care between the ages of 16 and 18 years. In some cases, patients may be treated at the same centre and, in other cases, they may need to transfer to another centre. There will be specific transition arrangements in each case which will include making sure you and your child / young adult are fully informed about what is happening and why and changes in consent to treatment. Once a child turns 18 the decision to consent to treatment rests solely with them as an adult (unless they lack capacity to make a decision).