In this day and age, millions of drugs and treatments have been designed in order to treat different illnesses. However, there are some fatal diseases that are “too rare to care about". Due to this rarity, these diseases, often due to unique genetic mutations, are not researched about, leaving their victims in the worst of conditions. Thankfully, novel drugs are being tested in order to fix these DNA mistakes.
One case of this would be when a specific drug was created for Mila Makovec, a little girl suffering from a rare genetic disease. Although this drug didn’t fix her mutation, it has stabilized her condition such as reducing her seizures and allowing her to walk again.
The effectiveness of this pill is due to the fact that it can be tailored to take the form of gene replacement, gene editing, or antisense. These molecules can erase or fix these unique genetic mistakes.
The only challenge associated with these new drugs are the costs and time spent making them. Since these unknown conditions are so rare, there are not many cases which means that the designing, manufacturing and testing time associated with these pills will only be able to fix a handful of people. However, doesn’t everyone deserve a chance at life?
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