PLEASE SIGN FOR THEO AND KATE !!

Update: DONATIONS ON THE PLATFORM DO NOT GO TO THEO BUT HELP SPREAD AWARENESS OF THE PETITION. THANKS EVERYONE 

Our son Theo was diagnosed with SMA (spinal muscular atrophy) type 1 in August 19th 2021 at just under four months of age. There is no cure and Theo has the most severe type. With this disease Theo's muscles become progressively weaker and affects not just his ability to crawl or hold his head up but also respiratory and feeding muscles.

He is recieving Sprinaza therapy but as he has such a severe type with only 2 backup SMN2 backup copies this is not the best treatment for him. Thankfully there exists another treatment for him but is out of reach in Ireland currently as is priced at approximately 2 million euros.

This other treatment is a gene therapy called Zolgensma. This treatment is available in the UK (and some other european countries such as Germany) but not available yet in Ireland, at least without paying the impossible price of the drug.

Zolgensma’s effect seems to go into every part of the body due to its systemic nature. So it works slightly better than Spinraza for motor skills and significantly better for respiratory and swallow functions. In particular Zolgensma will work better for Theo has only has 2 backup copies and these determine also the effectiveness of Spinraza. Based on the available data, it is incorrect to say that Zolgensma and Spinraza have the same effects for a SMA type 1 child which is what was claimed initially by the health technology assessment (HTA) in Ireland in May this year. Spinraza itself does not come cheap at 100,000 euros each injection, there is a loading phase of 4 doses and every 4 months for the rest of his life after that. Zolgensma is a once off gene therapy. More evidence on why Zolgensma is a better treatment, particularly when it comes to a low gene copy number, can be found in the review of Zolgensma by the European medicines agency.

The HSE in Ireland is current in pricing negotiations with Novaratis, the company that make the drug. We will not know the outcome of this negotiation until the end of the year and we want to ensure it is passed. It is the best treatment available and offers Theo the best chance at a normal life.

Help by signing this petition so that the HSE understand that by making this treatment available we can safely say that we will be helping Theo live the best life possible.

Thank you for your support it is appreicated more than you can imagine !!

Shane, Stephanie and Theo