The Harsh Reality of Rare Diseases: 95% Still Have No Approved Treatment

Let’s start with the numbers: over 10,000 rare diseases exist. Some are so rare that you might never even hear their names, and yet, they affect more than 300 million people around the world. Think about that for a second. That’s almost as many people as the population of the United States. But here’s where it gets interesting: 95% of those rare diseases don’t have a single approved treatment. Not one.

How is this even possible?

It’s not because they’re unsolvable mysteries that science can’t crack. The problem is much more frustrating. These diseases don’t get attention because they’re not “profitable” enough for big pharmaceutical companies. In other words, if you’re unlucky enough to have one of these rare conditions, you’re left to fend for yourself simply because treating you won’t make anyone rich.

A Broken System

The system we rely on for healthcare is pretty great at handling common illnesses. You break a leg, you get a cast. You need antibiotics, they’re there for you. But what happens when you’ve got something like AARS2 Deficiency, a disease that only a tiny percentage of people have? You get ignored.

If you think about it, rare diseases aren’t so rare. One in ten people has one. That’s one in every ten of your friends, coworkers, or family members. Yet, it’s those very people who are overlooked. Why? Because developing drugs is expensive, ridiculously expensive and pharma companies aren’t lining up to invest millions (or billions) into treatments for diseases that affect such small populations.

The result? People are left with nothing but hope, waiting for a miracle that never seems to come.

The Cost of Being Forgotten

There are children out there battling rare diseases, and every time their parents seek help, they’re told the same thing: “Sorry, no approved treatment. You’ll have to wait… if a cure ever comes.”

How does that feel? It’s not just a harsh reality, it’s a silent crisis. The tough part is knowing that science has made progress, but the resources to bring that progress to life just aren’t there. The system wasn’t designed to help those with rare diseases, and the sad truth is that the lives of these children are often viewed as too costly to save.

Curetopia BioDAO for Eradicating Rare Diseases 🌟

Curetopia is a BioDAO, and their work represents a collective push to change the way rare disease research is funded. Rather than following the traditional, slow path of waiting for big pharma to step in with funding (which rarely happens), Curetopia brings patients, families, and everyday people together to directly fund the research. Think of it as crowdfunding, but for life-saving breakthroughs. 🧬

In a major success, Curetopia raised $1.77 million to fight AARS2 Deficiency, a rare genetic disorder. Over 1,000 people came together to make it happen. It’s proof that when communities take charge, real change is possible.

And this isn’t just a hopeful gesture. Curetopia has already identified two potential treatments and is pushing forward to bring them into clinical trials. For the families dealing with this condition, it’s a light that hasn’t been seen from any pharmaceutical company. 🌱

A New Hope 🌍

This whole movement shows that rare diseases don’t have to stay in the shadows. The days of “waiting and hoping” are over. Curetopia is pushing the boundaries, challenging a system that’s been broken for far too long, and offering real solutions for people who have been ignored.

What does the future look like? Well, if Curetopia has its way, we’ll see decentralized clinical trials that move faster than ever, and research that focuses on repurposing existing drugs (because why reinvent the wheel when you can use what’s already out there?). And all of this is powered by regular people who care.

There’s no reason why 95% of rare diseases should be without treatments. The problem isn’t science, it’s the system. But that’s changing. 💡

With movements like Curetopia leading the charge, the future looks brighter for the millions of people affected by these diseases. There’s hope. There’s progress. And most importantly, there’s a community that refuses to let these diseases be forgotten any longer.

So, while the system may not care about rare diseases, we do. And together, we can Flip the Script.

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