We were lucky with diagnosis, we’re looked after by a muscular dystrophy centre of excellence. Our team is very positive, we spoke about the need of future ventilation the importance of physio. We were told that it was an exciting time for SMA. Although this was great and we usually have a super positive outlook it made our first PICU stay more difficult, everyone had expected us to have had that awful life expectancy conversation. We were told ‘you knew this day was coming’ err no, no we didn’t. I can guarantee Rexy would not be here if we weren’t in a hospital very experienced with SMA. First time we were in PICU and intubated we were taken into a tiny room and it was pointed out again that he was a type 1 that he was very lucky to have made it this far but we needed to prepare for the worst and call our family to come before it was too late. Rexy was 2 and a half! Once he started to look like he was going to pull through we were taken to the side by his respiratory consultant and told if we didn’t choose to go down the route of long term ventilation they could and most likely would refuse a future PICU stay, if he got ill again they would just make him comfortable and make sure he wasn’t in any pain. This is a baby they were talking about, that type 1 label makes| a big difference. We aren’t allowed to go to our local hospital, first thing we were told is a non specialist hospital will kill him. I drive past a brand new all singing all dancing hospital to get him to his hospital, we don’t go anywhere because we’re too terrified that he’ll get ill and won’t have access to a hospital that follows proper procedure. We aren’t just desperate for the drug, we’re desperate for a whole attitude change towards SMA.

I think everyone needs to be aware that the EAP is not a new thing, type 1 families have been fighting, screaming, shouting and bombarding doctors since summer. It’s globally only for type ls as it came from the success of the type 1 trial and what the company deemed as a need for compassionate use. This is a huge step for everyone as it’s giving the NHS (if they get their heads out of their arses) a chance to test the drug for themselves without paying for it, if it goes well and according to plan it will give Treat SMA huge ammunition and the EMA, NHS and NICE even less room to say no. The type 1 criteria is set out by Biogen and isn’t necessarily a type 1 diagnosis from a doctor, it’s basically showed symptoms at or before 6 months typical of type one (so never sitting).Type 1 families shouldn’t be made to feel bad or guilty that they might get the drug under EAP, there will be those that fit criteria that will not get it because there isn’t the capacity to administer it and they can’t get funded, at the minute it’s looking like it will be a postcode lottery. After our conversation this afternoon I had another type 1 mam PM me to say that’s how she felt too and that she didn’t like to comment on posts in the group or make posts as she didn’t feel comfortable. We’re a wealth of knowledge and experience use us but also be conscious that we face quite different attitudes to other types from everyone involved in care (which as I stated isn’t right but happens) most have a fight for the most basie help for their child, for doctors just to value their child’s life and see past that type 1 label.

Top image: Rexy’s self-portrait