Posted In: Health
Medical technology and practices have been advancing at an incredible rate over the past few decades, and one of the premier breakthroughs in the space is CRISPR. At a high level, CRISPR is a process that allows editing DNA sequences and gene function, which opens up a world of possibilities when it comes to developing new medicine and treatments. However, CRISPR as we know it today is not a perfectly precise tool, as it requires the rewrite of entire subsections of DNA or a genome. A new breakthrough in a joint study by Harvard and MIT has resulted in a new process called base editing, which gives researchers the ability to edit minute portions of DNA. In other words, if a single word needed changing in a paragraph, CRISPR would require rewriting the entire paragraph with one word changed while base editing would allow changing the specific word itself.
Amicus Therapeutics recently announced that they are seeking FDA approval for a new drug therapy designed to provide effective treatment for the rare condition called Fabry disease. What is Fabry disease and what does this rare condition entail? How does this new medication help control bothersome symptoms associated with Fabry disease? What is Amicus Therapeutics and what do they specialize in? The following information has been designed to answer these important questions.