We’re all familiar with what the news is saying about COVID and its effect, but how does it really affect rare disease patients and their families? We asked Jess Curran and Karen Morales to share the experiences they had when COVID made its way to their families.

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.

Gene therapy is the future of treating rare, genetic diseases that were previously thought to have no cure. CRD is breaking down what gene therapy is and how we are using gene therapy to develop therapies for neuromuscular diseases including Duchenne muscular dystrophy (DMD), limb-girdle muscular dystrophy subtypes, and spinocerebellar ataxia type 3 (SCA3).

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....

“So, how are they doing?” That is the most frequent question we get asked about our 2 boys (ages 7 and 10) who have been diagnosed with the fatal muscle wasting disease, Duchenne muscular dystrophy. There is no easy or short answer to this question because they are both currently doing well, but yet they aren’t because their bodies are slowly deteriorating. Thankfully Duchenne life hasn’t hit them very hard yet and most of their days are just like other kids, with a few exceptions.