Duchenne Muscular Dystrophy Will be Cured

You probably have seen the video.

An 11-year old boy walks down the stairs. Looks simple, even easy. Happens everyday in millions of homes, including yours. But his parents and sister are crying with joy. For them, it’s a miracle.

The boy has Duchenne Muscular Dystrophy, a defective dystrophin gene condition. It begins around age 4. Muscles lose strength in thighs, pelvis and then arms, primarily affecting the boys. Life expectancy is rarely beyond the teens. It’s genetic and can’t be done much to prevent or cure.

Until now.

There are clinical therapies in the pipeline and our young superhero had received one of those. A gene therapy (still in clinic) shot which edits the defected gene to correct the mutation leading to DMD. Once approved by the regulatory agencies, therapies like these will be able to provide the same miraculous moments to thousands of similar families.

Thanks to the evolving biopharmaceutical science, risk taking founders/investors and toiling scientists, we’ll witness more miracles like this in our lifetime.

I, for one, am looking forward to moments like this.