“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.

We previously lived in Central California and raised organically fed pork on pasture. When Kal was diagnosed with Duchenne...

Cure Rare Disease, in partnership with Charles River Laboratories, has successfully created 3 mouse models that harbor the humanized form of the dystrophin gene. Each model has a mutation that causes Duchenne muscular dystrophy that CRD is currently working to treat, meaning they can be utilized for critical in vivo studies to test the safety and efficacy of mutation-specific gene editing therapeutics. The success of this project paves the way for future therapeutics to be developed to treat rare and ultra-rare genetic disorders.