The Science of Rare diseases and orphan drugs

Greetings to my favorite science community online, StemSocial.

Today's blog is quite a special one because we're going to be discussing an issue I believe does not get discussed as often as it should.

This blog is intended to raise awareness for people suffering from Rare dieseases and if you have any knowledge contribution to make, I'll certainly appreciate it and respond in the comment section.

Now let's briefly discuss the science of rare diseases and orphan drugs.

Original Image Source by sasint from Pixabay

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I'm most certain that this topic might even seem new to most medical science enthusiasts because it's not as popular as topics about cancer or COVID. The reason is basically in it's name, Rare Diseases.

The real facts is that rare diseases, or orphan diseases, aren't that uncommon, even though each one affects only a small group of people. However when you begin to add them all up, you realize there are quite a few rare diseases out there, kind of like finding a bunch of hidden treasures.

Simply think of it like your very quirky aunt's collection of rare coins. Each coin is unique and interesting, just like how each rare disease has its own story.

But another thing is, not every doctor or medical scientist knows about these diseases. So, when you have one, it can be very difficult to have an expert treat or cure it.

Now here's a scenario to further explain my point.

Imagine you wake up one day feeling really strange, with symptoms you've never had before in your entire life. You then visit a doctor, and they look super confused about your symptoms.

Thern worse part is having to hear your doctor say, "I've never seen this before."

It's like you've entered a world of confusion. It has a way of breaking your spirit and any hope you might have had of recovery on your way to see the doctor.

You go from one doctor to the next, but no one seems to know what's going on. They try out different medications and non of them seem to work. The doctors simply cannot put their finger on what exactly you're faced with.

It's like being on a rollercoaster of emotions. You feel frustrated, and disappointed.

But then, one faithful day, you meet an expert doctor who listens carefully and manages to figures things out.

It's almost like a lightbulb moment to say Eureka. We finally discover that you have a rare disease.

Now, you finally realize that you're not alone and there's the possibility of treatment. You then join a close-knit group of people dealing with rare diseases.

You are able to share stories, give each other strength, and learn from one another.

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The Scientific Solution

Now here's where our scientific and medical solutions come in, The orphan drugs. These medicines are made just for rare diseases. These are definitely medications that are not popular and you might not have heard of.

Due to the rarity and complexity of rare disease, making these drugs isn't easy.

Finding enough people for tests can feel like a really big scavenger hunt that spans the globe. These are some of the unfortunate challenges that doctors face with rare diseases.

Another challenge which you probably would have guessed is that It's not cheap and common. This means that patients with financial scarcity or issues may find it difficult to have treatment even if the drug is available.

Making of the orphan drugs can be like saving up for something really special. However, when it means a chance at a better life or saving a life, I strongly believe it's worth every penny we would spend in manufacturing.

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Then there's also the part with all the rules and regulations around these drugs and treatments. There are still a few complexities around drugs made to treat these rare diseases. FDA approvals is also another hurdle since these diseases are a bit rare and complicated, getting approval from FDA can be a real challenge.

So yes, rare diseases and orphan drugs are a bit like a treasure hunt. It's got its challenges, and uncertainties. But in the end, it's a scientific journey filled with hope and discovery.

It also shows that when science, determination, and a little bit of luck I must say, come together, amazing things can happen and we can find solutions to complex issues.

To draw the curtains on my blog post today, the next time you hear about rare diseases and orphan drugs, always remember that it's not just complicated medical issues and experiments.

It's actually the story of real people, emotions, and the strength of human spirit.

Rare diseases might be rare, but the impact they have on people's lives is anything but rare.

I believe we need to come together and create more awareness for these kinds of dieseases. Due to their rarity, people suffer these diseases and some end up losing their lives. I believe medical science research with orphan drugs has the potential to treat and cure these dieseases.

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Thank you so much for taking the time to read today's blog. This blog was intended to raise more awareness for patients or people suffering from Rare dieseases. It's entirely opened for any question, comment or knowledge contribution you may have regarding the topic.

Have a lovely science day and catch you next time on StemSocial. Goodbye 👨‍🔬

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