top of page

Be Curious

"Kindness is more important than wisdom, and the recognition of this is the beginning of wisdom." ~ Theodore Isaac Rubin

What is Duchenne?

100% fatality rate

Caused by a change in the dystrophin gene

Characterized by a rapid deterioration of the muscles

There is no known cure

Dystrophin acts as a "shock absorber" that allows muscles to contract and relax without being damaged

Typically diagnosed between the ages of 3 and 7

Most boys with Duchenne are in a wheelchair by age 12

Occurs in 1 out of every 5,000 boys

Dystrophin is critical for maintaining muscle cell structure and function











Imagine a world where genetic diseases could be reversed. We do every day and have funded over one million dollars of groundbreaking research! Our main focus going forward are on two promising therapies – CRISPR and Gene Therapy. 

After decades of slow progress in the fight against Duchenne, the FDA approved the first Duchenne drug ever on Sep 19, 2016. That was a big day in the fight against Duchenne. The drug, called eteplirsen, has already proven to be a game changer for some patients, however, since Duchenne presents itself with slight variations, this drug only applies to about 13% of the Duchenne population.



SINCE 2015

Another game changing treatment called “gene therapy” or “gene editing” is advancing rapidly as a viable

treatment. With gene editing, the goal is to introduce a portion of the corrected code for dystrophin, providing the cell with the recipe to produce some of the required dystrophin on its own, and thereby reducing muscle damage and promoting necessary muscle repair.

Serapta Therapeutics, Pfizer and Solid Biosciences began micro dystrophin clinical trials in 2018 and early reports from all three are encouraging. Patients experience improvements in time to rise from a sitting position, time to climb 4 stairs, and time to walk 100 meters. These improvements are particularly impressive given the fact that in the normal course of Duchenne, the patient experiences a steady decline in these baseline functional tests. Additionally, reports indicate that patients are experiencing a robust expression of the micro-dystrophin gene.

Conner was the very first patient dosed with Pfizer's micro-dystrophin gene therapy. All of his functional tests improved for a period of about 2 years, and then began to regress. We observed an initial impressive improvement in his stair climbing ability, with a decline with respect to the speed of his assent a couple of weeks later, and then a leveling off in speed. He has since experienced a slow decline in his stair walking ability, and is not able to navigate more than a few stairs. The stair climbing speed and functional abilities decline post infusion were expected by many researchers as newly formed muscle from the natural growth process does not contain the micro-dystrophin gene therapy that was given to Conner initially. We look forward to a time when re-dosing of gene therapy may be possible.

Funded Research


Kindness Over Muscular Dystrophy has funded exciting new advancements in CRISPR genetic research that have the potential of fixing faulty genetic code in a DMD kid’s muscles. This enables those damaged muscles to stop deteriorating and to function better. CRISPR is the gold standard for a cure – fix the flaw in the genetic code, and stop the muscle damage. 

Gene Therapy

Gene Therapy is the next best thing to CRISPR, and Kindness Over Muscular Dystrophy has funded research that has produced ongoing clinical trials of this therapy. Kids show muscle function improvement for a couple years or so, then a leveling off, and then decline. Re-dosing would be a great solution, but the problem is that re-dosing can cause the immune system to overreact in a severe life-threatening allergic fashion. KOMD is in the process of funding re-dosing research to circumvent this issue. 

Benevolent Assistance

A portion of the money we raise at Kindness Over Muscular Dystrophy goes to help those families who struggle with the financial burdens that a complex medical disease like Duchenne Muscular Dystrophy brings to their tables. The costs of wheelchairs, stair lifts, accessible vans, and accessible improvements to homes can be astronomical. To help offset these financial hardships, we at Kindness Over Muscular Dystrophy have granted benevolent assistance funds to the Jett Foundation and to Team Joseph to distribute to families in need.

Our funding of research and benevolent assistance would not be possible without your generosity. Because you care, Kids with Duchenne have hope for a healthier future in a more accessible world!

From the entire KOMD family, and from all of those whose lives are impacted by Duchenne...


bottom of page