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

Talk to your / your child’s medical team:

• Are they aware of where you / your child may be able to access the trial you’re thinking about?
• Are there any clinical trial or other results so far and, if there are, what do they suggest might be the outcome for you / your child?
• Does your health / the health of your child pose any risks of travel for the trial?
• In their view how close should you /your child be living to the trial Centre and for how long – to enable follow-up and monitoring, and in case of any complications?
• How would having a trial provided in this way impact on your / your child’s care and support from their current ‘home’ team?
• If the trial results seem successful, what happens next if the treatment isn’t funded in the UK

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

• Their eligibility criteria and referral process – how they would assess you / your child for this
• How often would you need to attend and for how long would the trial continue?
• What follow up is required at the overseas centre?
• Where you would stay during and after the trial?
• How much would it all cost – the travel, accommodation and would any of this be funded by the overseas centre?
• What happens when the trial stops?

Discuss with your family / support network about:

• How you’re going to get there. If you’re flying you may find information in the Living With SMA section helpful e.g. the Plane Travel section on this page.
• Costs that won’t be covered by the trial sponsor. These may include: 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, care for others at home, care for pets at home
• Managing this commitment practically, emotionally and financially – perhaps on an ongoing basis

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

For us as a registered UK charity, web-based fundraising, such as crowdfunding, presents 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.

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.

Ask your / your child’s medical team.

The pharmaceutical companies that we are aware of that are actively working on treatment for SMA:

• Spinraza™ / nusinersen
  Biogen: MedInfoUKI@biogen.com
• Zolgensma™ / onasemnogene aboparvovec
  Novartis: medinfoemea.gtx@novartis.com
• Evrysdi™ / risdiplam
  Roche: medinfo.uk@roche.com

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.