Always for the Children

JFF Blog

Wed, Dec 06, 2017

A Reason to Celebrate


Category: General 


Dear Friends and Family,

For several years now, every December we come to our investors to report on the happenings of the year and we call on you to continue funding our goals. For the past several years we’ve shared that we’re closer to a cure than ever before. Last year, we were hopeful that clinical trials would begin soon. It is my great honor to share that on April 3rd, 2017 the FDA awarded Investigational New Drug Application (IND) for MTM gene transfer therapy, and the week of September 20th, 2017 was the commencement of clinical trials.

As I sat thinking of the first child receiving the first dose of this drug, I imagined the intense emotions these parents might be feeling, knowing their child has potentially been given the gift of hope.

I then began to think back over the last 22 years of my life, envisioning it like a movie playing across my field of vision. Hope, fear, vision, fortitude, grief, and perseverance mingled together around a central theme: finding a therapy for Myotubular Myopathy. I thought about our son, Joshua, as a beautiful and frail infant, hooked to numerous machines in the NICU. That first year of Joshua’s life  was a blur, resuscitating Joshua more times then I could bear, learning his diagnosis, and starting the foundation. I beamed with pride as I thought about our researchers, one by one, who have dedicated their professional lives motivated by their altruistic mindset in moving this research forward. I smiled as I remembered Nibs, a brown chocolate lab who was the matriarch of our dog colony. Tears rolled down my cheeks as I relived Christmas Eve 2010, the day our son lost his battle to this tragic disorder. Losing him brought our family to its knees, but that seemingly insurmountable loss gave us more resolve and fight, and we vowed that no parent should face the unfathomable loss of a child.

Through all the memories, there is a path of connection from one success of research to the next. There is also a path of connection from family to family. Like a snowball that turned into an avalanche - our life is full of families all over the world who share our story. The original 50 known cases have grown to a projected 5,000 to 8,000 affected children. Over the years, one of my primary roles has become patient advocate. There is not a single day that goes by that I’m not in contact with a family in one capacity or another. Twelve of these families will participate in the clinical trial, and the need for patient advocacy is greater today than ever before.  As crushing as it is to share this, nine of our families buried their children this year, four in the last three months alone.

Your investment continues to support The Joshua Frase Foundation’s mission and goal. You’ve helped mothers and fathers pay for funeral expenses and put tires on a van needed for transportation for their handicapped child.  Your donations supplied medical equipment and adaptive devices to meet the unmet need of insurance. You’ve helped families who have to choose between work and their children to keep their lights on. And, of course, because it’s in our DNA to continue searching for anything viable that will be beneficial for the lives of our children - we continue to support cutting edge research initiatives.

We ask that you continue to come alongside us in this most crucial time, as we continue the foundation’s mantra to “make a change in the lives of the Joshua’s of the world.” At some point on the journey, circumstances have forced us to make a choice; we could let this make us, or we could let this break us. One of those circumstances was the loss of our son. Quite frankly, that is the sweet spot. It is those decisions that foster change. Change happens when we take the hand we’ve been dealt, and we shuffle the cards. Change happens when we look at what is, and re-focus on what can be.   

The kind of change that adds beauty and hope to the world around us isn’t easy to come by. It requires effort, dedication, patience, perseverance, courage, hope, and flexibility. It requires the ability to remain unwavering in the face of criticism, ridicule, and despair. To bring change and hope to the world requires that we trust our gut instinct when we have no logical reason to walk the path that we’re walking. It requires  that we get comfortable being uncomfortable.

We are humbled to share that the path our foundation has forged is being used for other indications, and we look forward to the day when this disease is eradicated from the list of rare diseases without a cure.

Many Blessings to you and your family this holiday season from the Joshua Frase Fnd. 
Alison, Paul, Isabella and Joshua Frase 

If you have not had the opportunity to view our short documentary, "Brick in the Wall," is a poem written by Joshua in his 9th grade English class.  please click on image below: