Tuesday, January 23, 2018

A #FightingForMaya Update: What if you had to?

Several months ago, I awoke in the middle of the night.  I wasn't really startled, but I just woke up and couldn't fall back asleep. Like too many of us, I immediately reached for my phone and checked if I had missed anything during the last few hours of slumber.

As some point, I realized that a friend (another rare disease dad) was awake and online.  Perhaps I was intruding, but I pinged him and asked how things were going.  After a few rounds of back and forth, I asked why he was up (he is a couple of hours ahead of us).  To my surprise he answered with painful honesty... he was wondering if he had done enough.  Had he missed anything in his pursuit to save his children.

It was brutal.  It was honest. It is a common struggle within the rare disease community.


There really isn't a rare disease playbook.  No one person or organization has the answers.  The list of things that can or must be done is simply overwhelming.  I've found that most parents pick one or two areas of expertise (e.g., legislation, research, fundraising or caring for their child) and drive hard in that particular direction.

In the face of overwhelming odds, it seems to be the best possible answer.


Last week, I had the absolute pleasure of visiting Agility Clinical and sharing Maya's story.  Our friend's sister works at Agility and she has followed Maya's story over the last couple of years.  When Agility scheduled their 2018 All Hands Meeting, she asked if I would be interested in sharing a bit more about our family's story.

Agility Clinical is focused on the rare disease community and provides full clinical trial services from study design through to regulatory submission for marketing approval.  They are good people.

Below are a couple of pictures from the talk.  Thank you Laurie, Agility Clinical and Mark for helping us share our story.


The question I receive most often is "How is Maya?"Its a tough question to answer, but she is doing well.  Without treatment, we believe that Maya's gains would be non existent and her progression would be far worse.  Although we don't have a cure, we do feel that we have bought more time.Maya works very hard everyday to improve her walking and balance.  She can't walk independently and depends on a wheelchair to cover long distances. However, she shows such conviction and drive to regain the ability to stand and walk.  It is inspiring.


"What if you had to?" is something that my favorite spin instructor asks of the class at those most critical of moments.  Those moments when 99% of your body and mind says give up and only 1% is pushing you forward.

Each day we are fortunate enough to witness Maya and so many other Rare Disease patients and families answer the question... What if you had to?

Thank you for your continued love and support.


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