Hope for Helena

Hope for Helena


Everyday when I would wake up I would check social media and long for the day when I would read that Biogen/Ionis (the pharmaceutical companies developing the drug Nusinersen) have filed for approval of their drug….

Yesterday that dream became a reality!

It has been announced that in the coming months these pharmaceutical companies plan to file for approval of the drug Nusinersen for use by children with Type 1 (Helena has type 1) spinal muscular atrophy. This means that the drug could possibly be approved for use for type 1 children as early as next year. But excitingly the pharmas have also announced that in conjunction with this application for approval that they will be ending the current trial in type 1s and the placebo arm of another trial containing older type 1s which will allow for an expanded access program for other children suffering the condition that weren’t able to participate in trials. There will be inclusion criteria for access to this and it will only be an option for countries that have expanded action program options (which the UK does), but this is the best hope we’ve had yet that Helena and many other children with type 1 SMA might be able to receive treatment this year!

The inclusion criteria for this expanded access will be announced in the coming weeks and if she meets the criteria it’s difficult to predict the affect that receiving Nusinersen will have for Helena at this point. We know that she won’t be cured by it because she has already had SMA for 17 months now, but the progression and course of the condition is individual in each case and so their is still some hope she may see some improvements, but even more hope that this drug will prevent further progression of her SMA. There are also many more upcoming treatment options in the pipeline and it’s generally agreed that the best results will be seen with a combination of treatment options, but this is a great first step on that path.


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