Information for People Living Outside the UK

As a small UK charity we regret we do not have the capacity to respond personally to individual enquiries when the person who has SMA is not resident in the UK. We hope the information on this page may answer some of your questions and be helpful to you.

Q 1. Where can I find information about SMA and treatments for SMA

A. We provide information about SMA  including information about treatments which you are welcome to read, though please bear in mind that these pages are written for those living in the UK and with access to UK health services.

Q 2. We’re thinking about applying to access a treatment that is available in another country. Have you any advice?

A. There are lots of considerations. We suggest you would need to:

Talk to your / your child’s medical team about:

  • The clinical trial results and what they suggest might be the outcome for you / your child
     
  • Your health / the health of your child and any risks of travel for treatment
     
  • Their view of how close you should be to the treating Centre and for how long this should be, in case of complications arising from the treatment
     
  • Any difficulties there might be with having a treatment provided in one place and ongoing care in another. For example, would your own Centre still be able to offer you / your child the Standards of Care (scroll down on this page) and support needed?
     
  • If they are aware of where in the world you / your child may be able to access treatment

Talk to the overseas centre you are considering about all of the above plus:

  • Their eligibility criteria and referral process - how they would assess you / your child to see if you / your child are eligible for treament
     
  • How often you would need to attend and for how long treatment would continue
     
  • What follow up is required at the overseas centre
     
  • Where you would stay during and after treatment
     
  • How much it would all cost – the treatment, the travel, accommodation and whether any of this would be funded by the overseas centre

We suggest you consider and talk to your family / support network about:

  • Travel insurance to cover your child’s / your and any travelling companions’ health and other needs
     
  • Managing this commitment practically, emotionally and financially – perhaps on an ongoing basis

Q 3. What are the International Standards of Care for SMA? (including in different languages)

​A. Whether or not you / your child is able to access  a drug treatment, you may find it helpful to know about the International Standards of Care (SoC) for SMA. Tnese describe the intervention and care recommended for best quality of life. Many families find it helpful to discuss these with their medical team.

You can download a copy through the link here. If you scroll down the page you’ll come to a section of flags which link to the Family Care Guides in different languages.

Q 4. How can we find Patient Support Groups in other countries?

​A. Follow the link here – and scroll down to the section ‘Patient Organisations’.

Q 5. We’re thinking about raising funds to access a treatment in another country. Have you any advice?

A. We understand people’s desperation, but we are concerned by the uncertainties and pressures this potentially creates for families and adults and for the clinicians who are asked to and want to treat them if they can do so safely.  Things to consider include:

  • If a treatment is repeated or ongoing, how much money needs to be raised before treatment starts?
     
  • How much money will continue to be needed to carry on with the treatment
     
  • Potentially treatment may be stopped abruptly if funding runs out.
     
  • If a treatment is a ‘one off’, regular and close follow up may still be needed.
     
  • In addition to the actual cost of treatment, consider additional costs, such as travel, food and accommodation, travel insurance to cover your child’s / your and any travelling companions’ health and other needs, unexpected delays or extension of time away

Q 6. Can you, or another UK charity, help us with our fundraising or share our fundraising campaign through your website / social media?

A. We regret that we do not know of other UK charities that are able to help families from overseas with fundraising for treatment

For the following reasons we regret we cannot help with or promote any personal fundraising campaigns.

In our view, no family or adult should have to find funds for treatment either through fundraising or their own private means. This is not how treatment should be allocated.

For us as a registered UK charity, web-based fundraising, such as crowdfunding, present potential issues:

Though most people are genuine and will later post evidence of the outcome of their campaign, web-based platforms have no requirement for this and there’s no accountability to the people who’ve made donations as to how the money is actually used. Sadly, these options are potentially open to abuse.

We don’t have the resources to undertake these very important checks. Nor would we wish to judge which campaigns are ‘worthy’ and which less so.

In our view, regulatory authorities and pharmaceutical companies need to find ways for treatments to reach all those in need for whom there is  clinical evidence that this is an appropriate treatment.

Q 7. We’re thinking of contacting the pharmaceutical companies to ask if there are any countries where I / my child might be able to access treatment. Have you any contact details?

​A. These pharmaceutical companies can be contacted by email:

Spinraza™ / nusinersen

Zolgensma™  / onasemnogene aboparvovec

Evrysdi™  / Risdiplam

Q 8. We’re thinking about applying to ‘swap’ treatments or access a second treatment  ‘in combination’ with the current treatment, are there any issues with this?

A. Talk to your medical team about whether this is a possibility and, if it is, how this should be managed and what risks this may pose for you / your child.

Q 9. There’s a treatment that we’ve heard about, but you haven’t been reporting on it. What do you think about us going for this?

A. It is important to be aware that there are unauthorised or unlicensed ‘clinics’ in various parts of the world offering unconventional treatments, usually for large sums of money. The internet contains sophisticated websites which look appealing but make unsubstantiated claims about alternative treatments. It is critical to get an objective view from your clinical team before contacting such clinics or considering travelling abroad.

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.

Using an untrialled / unapproved combination of treatments presents similar dilemmas for clinicians.

Q 10. We’re thinking of applying to take part in a clinical trial. Have you any advice?

​A. The information we have about clinical trials is taken from the international website here where you can search for globally registered trials by drug treatment or condition name.

There are many patient registries that have been set up across the world that specifically collect data from patients with SMA. These sometimes share information about clinical trials for SMA. If your country has a national SMA Patient Registry, you may wish to sign up to it here.