Skip to Content
Our sites' mission is to document and discuss issues of the day regarding Muscular Dystrophy and how we are working to make all of our sons and daughters lives better.

Gene Repair Therapy (PNA-ssODNs)

As a parent of children with Muscular Dystrophy, or any genetic mutation for that matter, I must say I am ecstatic to hear the latest news in that field. Although in the very early stages of research, the initial results have shown very encouraging results. In the latest edition of Quest magazine and the Oxford Journal, there is discussion of an emerging therapy called Gene Repair Therapy.

“Look at me…I’m Nobody!”

As many of you know, we have two young boys with Duchenne muscular dystrophy. We live in a neighborhood with many children and our boys play with all of them to the best of their ability. My oldest boy still likes to ride his bicycle. We had gone through the process of getting him a small mountain bike with selectable gears. We set them to an easy setting, allowing him to pedal on flat ground with his reduced leg strength, but still with significant difficulty going up even the slightest inclines. As he’s grown older, his friends now all ride their bikes much faster than him.

Syndicate content