Unlike traditional methods for developing therapeutics for common and (relatively) rare diseases, ultra-rare disease drug development requires a different approach and mechanism in light of a limited ability to commercialize and even reach BLA/NDA approval once clinical trials are complete....

In the media, people with disabilities often aren’t featured as main characters, if they are included at all, so we wanted to share some kid-friendly shows and movies to help teach kids about acceptance and inclusion. Representation in movies and TV shows is important because it shows people, especially younger kids, that not everyone will look or act the same way and that it is important to be accepting no matter what. 

An Interview With Seda Filenko As one of the first families dosed with gene therapy, what were some of your thoughts, feelings, hopes, fears, anything you really considered as you made the decision to enroll in the trial? The idea…

CRISPR mediated exon-skipping is a method of genome editing that can be used to develop life-saving therapeutics for people with rare and ultra-rare genetic disorders. CRD is explaining the science behind these technologies as well as how they are the key to treating these diseases.