Joseph’s journey with Duchenne Muscular Dystrophy

Hi everyone, we are the Mackey’s- Jake, Angie, and Joseph. Jake is a fire fighter for NKC and an Army veteran. I am a manager for ComminityAmerica Credit Union. Joseph is a sweet, lovable, silly, and amazing 5 year old who just wants to be like all 5 year olds!

As you may know, our son Joseph was diagnosed with Duchenne Muscular Dystrophy. It has been difficult to navigate as a family but we have definitely become stronger because of it.

We have recently started Joseph on steroids in hopes of getting him into a clinical trial. Which, unfortunately is not going as smoothly as we’d hoped and Joseph didn’t meet the criteria for Exxon skipping (being .6lbs under weight at the time). So now we’re left waiting for 6 months plus waiting for a trial. All the while,

Josephs muscles are deteriorating.

Steriods are the only “treatment of DMD” however steriods have such a negative effect for long term use and there is no cure! Not to mention behavioral effects and weight gain (which is very hard for a DMD kiddo already struggling physically with added weight). Prolonged use of steroids also has negative effects on bone health, kidneys, and his liver function. Joseph has been on Prednisone because there has been no other choice.

We decided to switch to Agmaree, a new steroid on the market with less side effects and safer for long term use. The cost- $10K per BOTTLE!!!! What’s more- our insurance has denied it because, as they say it’s “not medically necessary”. We are truly at a loss!

We were also hopeful for Gene Therapy, which was approved by the FDA however, Joseph’s antibody levels are too high to receive the treatment. Meaning his body would reject it and this could be life threatening.

Being a parent of a kid with a rare and terminal disease can be frustrating to say the least.

This is why we’ve decided that it’s time to take Matters into our own hands and advocate for our son!!

There are Drs who specialize in DMD across the US that we are desperately trying to connect with.

With this, things like the cost of travel, continued medications and supplements, and paying out of pocket because our insurance isn’t approving it, and without a clinical trial as an option, it’s adding up fast.

The cost alone for gene therapy is $3.2 million. Even with insurance the out of pocket is unimaginable and not something an average family like ours can afford.

My husband is a fire fighter and an army veteran, we deserve better than having to fight Drs, insurance, and advocate alone!

We are asking for your help to lessen the burden on our family to fight for our son’s life. Not having to stress over medical bills that have stacked up, and will continue to stack up, would allow us more time to focus on a treatment option for Joseph. Every little bit makes a difference.

The only way we can move forward in strength is because of people like you and your support. We are truly grateful to have you join us in the fight for Joseph’s life.

For those who don’t know, Duchenne Musuclar Dystrophy (DMD) is a genetic disorder. DMD causes progressive muscle degeneration and weakness due to the alterations of a protein called dystrophin. With DMD, dystrophin does not continue to produce and Over time, boys with DMD like Joseph will loose all muscle function and are likely in wheelchairs by the age of 10. The life expectancy of a DMD kiddo is about 20 years old without treatment.