Pondering the Possibilities of the Future

A few columns ago, I wrote that I no longer was pursuing Spinraza (nusinersen). I still have no qualms about that decision, because we have a number of potential treatments that are slowly making their way to FDA approval, including risdiplam. This is an investigational treatment I am very excited about.

I don’t usually get excited when potential SMA treatments come out. Often, as with Spinraza, approved treatments are incredibly expensive or not meant for every type of SMA.

Risdiplam is currently in clinical trials. From what I understand, developers Roche and Genentech, along with PTC Therapeutics and the SMA Foundation, hope for a decision on FDA approval in May. It is an oral treatment, unlike Spinraza, which is delivered via spinal injection. Not all of us can get spinal injections, sometimes because of spinal fusions to correct scoliosis.

So, something like risdiplam is a lot more accessible because it’s a liquid. That would be even easier, as I wouldn’t have to swallow a pill. 

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It’s nice to know there are treatments under consideration that have more potential for other types of people and situations. It makes me excited to follow the journey that risdiplam takes, and to see whether it will receive FDA approval and what that would look like for insurance plans across the world. Also, what would the price be, as these things tend to be less expensive than medical procedures? And finally, it would be exciting to see for whom it works for in the end.

I believe that with all the results we’re seeing from trials, it won’t take long for it to be approved by the FDA — but that is entirely my opinion.

When I first found out about Spinraza, it took only a year for it to be approved in most places. In the grand scheme of things, that really isn’t that long. When you or someone in your family is sick, you want these things immediately. I understand that.

Soon I’ll be 23, and so far, one treatment is on the market for what I have, and two are in clinical trials with promising results. One is promising for my situation. I think that’s wonderful, considering that there may be a future in which kids will get to be kids instead of having to grow up quickly because of a disease. 

There may even be a possibility that in a couple of years I won’t be as sick or I won’t progress as quickly. 

That’s a beautiful thing to look forward to. 

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Note: SMA News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of SMA News Today, or its parent company, BioNews Services, and are intended to spark discussion about issues pertaining to spinal muscular atrophy.