These sections of our website are probably most useful. But, they are written for people in the UK with access to UK health services.

About SMA >

Recently Diagnosed with SMA >

These sections of our website are probably most useful. But, they are written for people in the UK with access to UK health services.

Nusinersen (SPINRAZA™) >

Risdiplam (EVRYSDI™) >

Onasemnogene Abeparvovec (ZOLGENSMA™) >

​The Family Guide to the Standards of Care for SMA > sets out what care anyone who has SMA should have, wherever they live. It has been translated into 10 different languages. You can download copies.

There is an English website version of the Family Guide >.

This care is important with or without drug treatment.

We suggest you discuss these standards with your doctor.

To get free NHS healthcare in England your child will need to be "ordinarily resident" in the UK. This means you must be living in the UK on a lawful and properly settled basis for the time being. You may be asked for evidence of this.

For a detailed definition of what being ordinarily resident means, see the GOV.UK guidance.

You or your child will also have to meet the eligibility criteria for the treatment. You can find out more in this pages:

Nusinersen (SPINRAZA™) >

Risdiplam (EVRYSDI™) >

Onasemnogene Abeparvovec (ZOLGENSMA™) >

If you are thinking of moving to the UK, you can find further advice in these pages:

Moving to England from EU countries or Norway, Iceland, Liechtenstein or Switzerland >

Moving to England from outside the European Economic Area (EEA) >

We suggest you:

Talk to your doctor about:

• What they know about the treatment.
• How they think it might help you.
• Your health and any risks of travel for treatment.
• How close you need to be to the treating hospital and for how long. In case of complications from the treatment.
• Any risks there might be having a treatment provided in one place and ongoing care in another.
• Where in the world you may be able to access treatment.

Talk to the overseas hospital about all of the above, plus:

• Their eligibility criteria and referral process.
• How they would assess you.
• How often you would need to attend.
• How long treatment would continue.
• What follow up is needed at the overseas hospital.
• Where you would stay during and after treatment.
• How much it would all cost:
  • the treatment
  • the travel
  • accommodation
• Would any costs be covered by the overseas hospital.

We suggest you also talk to your family and supporters about:

• Travel insurance to cover your health and other needs.
• Travel insurance for anyone going with you.
• Managing the commitment practically, emotionally and financially. It may be on an ongoing basis.

We understand people’s desperation. But we worry about the pressures this may create for families and adults. We also worry about the pressures on the clinicians who want to treat them.

As well as thinking about what we suggest in question 6,  we suggest you think through:

• If a treatment is repeated or ongoing:
  • How much is needed for it to start and then continue?
  • What happens if money runs out and it then stops?
• If a treatment is a ‘one off’:
  • you may still need regular and close follow up.
• As well as the cost of treatment, you will have other costs:
  • travel
  • food and accommodation
  • travel insurance to cover your health and other needs
  • travel insurance for anyone going with you
  • unexpected delays or a longer time away

Sorry, but we do not know of any UK charities that are able to help families from overseas fundraise for treatment.

We regret that we cannot help with or promote any personal fundraising campaigns.

It is our view that:

• No family or adult should have to fund treatment through fundraising or their own money. This is not how treatment should be allocated.
• Web-based fundraising, such as crowdfunding presents problems:
  • Most people are genuine and will later post evidence of the outcome of their campaign.
  • But web-based platforms do not require this.
  • There is no accountability to the people who have made donations as to how the money is actually used.
  • Sadly, this all means these options are open to abuse.
  • We do not have the capacity to undertake these very important checks. Nor would we wish to judge which campaigns are ‘worthy’ and which less so.

• Regulators and drug firms must find ways to ensure that effective treatments reach those in need.

These drug companies can be contacted by email:

• Spinraza™ / nusinersen
  Biogen: MedInfoUKI@biogen.com

• Zolgensma™ / onasemnogene aboparvovec
  Novartis: medinfoemea.gtx@novartis.com

• Evrysdi™ / risdiplam
  Roche: medinfo.uk@roche.com

Patient groups for countries in Europe are listed on the SMA Europe website >.

Talk to your doctor to find out if this is possible.  Ask how it would be  managed and what risks there may be for you.

Some parts of the world have unlicensed ‘clinics’ that offer unconventional treatments. This is usually for large sums of money.

They may have a website that looks good. But the claims they make about a treatment have not been proved.

Always talk to your doctor before going any further.

If a clinician is asked to treat a patient ‘off label’, for example a patient with a clinical diagnosis of an SMA Type for which there is no licensing authority, they are potentially liable if something goes wrong.

Clinicians are also potentially liable if they use an untrialled / unapproved combination of treatments and something goes wrong.

​You can search for a drug treatment on this website of globally registered trials >

Patient registries that collect information from patients with SMA sometimes share information about clinical trials. You can find out if your country has a national SMA Patient Registry >