There’s a part of this journey I don’t think many people talk about. Not the fear, not the uncertainty, not even the clinical science — but the isolation that comes with trying to find treatment for a rare condition like Spinal Muscular Atrophy (SMA) when information and access feel locked behind doors no one will open.
I’ve been asking. Reaching out. Trying to get answers. Trying to ask for help. And most of the time… I’m ignored.
This isn’t about cost — not yet, anyway. Right now, it’s about access to information and being heard.
The Promise of New Treatments — And the Silence Around Them
Recently, a new treatment called Evrysdi (risdiplam) was registered in South Africa for the treatment of SMA.
This is huge. It means there’s now an oral medication available here that wasn’t before — something people with SMA across all ages can potentially take at home. (mdfblog.org.za)
But here’s the thing:
- What does this really mean for people living with SMA here today?
- How do we access it?
- Who can prescribe it?
- Who pays for it?
- Is public healthcare ready for it?
- Are doctors informed enough to discuss it clearly with patients?
Because I’ve asked — and so far, I haven’t gotten comprehensive answers.
So What Is Evrysdi?
Let’s break this down so we’re clear about what the science says — because that’s exactly the kind of information we’re supposed to be able to have.
Evrysdi is a treatment designed to increase levels of the survival motor neuron (SMN) protein — the very protein that people with SMA are missing because of genetic mutations. This protein is vital for the health of motor neurons, which control muscle movement. (Drugs.com)
Unlike some treatments that require spinal injections, Evrysdi is taken orally, once a day, either as a liquid or a tablet for those eligible. (Roche)
It doesn’t cure SMA, but it modifies the course of the disease. It helps the body produce more of what it’s missing. (Drugs.com)
That’s huge. But here’s the heart of it:
None of this matters if people can’t find out about it, understand what it means for them, or get it prescribed — because no one is talking about it.
Why I Feel So Alone
I’m not just trying to find medication — I’m trying to understand my options. I’m trying to connect with professionals who know this subject deeply. I’m trying to get proper guidance.
Instead, when I ask:
- I get radio silence
- I get vague answers
- I get redirected
- I get ignored
And the worst part isn’t the lack of answers — it’s the sense that I’m the only one fighting to even find out if the answers exist.
It feels like every time I reach out, I’m shouting into a void.
The Bigger Questions We Deserve Answers To
This experience has made me wonder:
❓ Why is so little communicated to people living with SMA about new treatment options like Evrysdi?
❓ Is it because the system doesn’t know how to handle new therapies yet?
❓ Is it cost? Bureaucracy? Lack of education? Or something worse — indifference?
❓ Where do people like us go to get real, clear, honest information?
❓ If a treatment exists, why does it feel like nobody wants to help us access it?
❓ Who is responsible for bridging these gaps — and how do we hold them accountable?
Because this isn’t just about medicine — it’s about dignity, inclusion, and being worthy of support.
You Are Not Alone — But It Often Feels That Way
If you’re reading this and you’re also trying to figure out:
- how to access treatment,
- how to talk to your doctor,
- how to get information before someone else asks about cost,
- or how to be treated with respect and seriousness…
I see you. I feel that frustration deeply.
You shouldn’t have to fight for answers like this on your own.
Let’s talk. Let’s share what we know. Let’s build a community of informed voices, because no one should face this alone.
If this resonates with you, I’d love to know:
💬 What has your experience been like when asking for information about SMA treatments?
💬 Have you or a loved one been offered Evrysdi? What happened next?
💬 What questions do you still need answers to?
💬 What would help you feel less alone right now?
Let’s start that conversation.
