For the longest time I have been deciding what my first article for this blog should be about. Since I’m interested in almost anything that comes into my hands and brain (as every proper science enthusiast 😊), it was not easy to decide on just one topic. But then it came to me. Not too long ago I watched a movie that definitely didn’t leave me apathetic, so I came to the logical conclusion to share it with you. “A Beautiful Mind” is a perfect example of science in everyday life, so I wanted to delve into it more closely and in as much detail as possible. Before I start the analysis of this wonderful movie, I should take a moment and warn you that there are going to be spoilers along the way, so if you want to watch the movie first, I highly recommend you do so.
Friedrich’a ataxia (FA) is a progressive neurodegenerative disease that affects 1 in every 50 000 people worldwide. Therefore, it falls under the umbrella of rare diseases. The thing with rare diseases is that it’s hard to get funding for researching their pathophysiology and possible therapies (ergo the name “orphan drugs”). However, with the recent rise of gene therapy, more and more private investors put their money towards finding a cure for 1 in 50 000 people. So don’t be misled by the title of this article – FA is still a rare disease, but its popularity among research groups and institutes has been growing for the past few years. The main reason for such blooming is the emerging field of gene therapy.
What is a clinical trial and how (why) does it work?
A clinical trial is a study conducted on human volunteers to investigate a variety of questions on a treatment/intervention tested:
- is the treatment/intervention safe?
- does the treatment/intervention work?
- does the treatment/intervention work better than what is already available (if there is a similar treatment/intervention)?
Co-author: Mario Borna Mjertan
Greetings, S3 blog reader! Our interactive quiz strikes again! As with our first quiz (check it out if you haven’t already), the questions are varied, this time from literature to pharmacology. Once again, don’t worry if you are not familiar with some terms, try to answer anyway.
Each question has only one correct answer. When you choose the answer, you will see whether you got it right and get an additional explanation. Beneath each question there is a link to the text from our blog that deals more with its respective topic. Have fun!