Saturday, August 12, 2017

A #FightingForMaya Update: Celebrate the Small Stuff

So it has been a bit of an intense week.  On Friday, we celebrated Maya's 22nd infusion.  I know, 22 is an interesting number to celebrate, but for our family it has turned out to be a really important number. A number worth celebrating

You see, yesterday was our first "commercial" infusion.  Maya officially, graduated from the compassionate use program for the enzyme replacement therapy and is now receiving replacement enzymes on a commercial basis.  "Graduation" means many different things, including, fewer vitals, no more over night stays, insurance pays for the treatment and hopefully less uncertainty.  The year leading up to this moment in time has been a grind, but we made it.

This week, exactly one year ago, our family made our first trip to Columbus, Ohio.  We went to Columbus so that Maya could be screened for admittance into the compassionate use program.

Except for a battery of tests, we really didn't know what to expect of the appointment.  For the most part, Maya had a really tough time.  She isn't that great with new people and during the appointment, she had to cooperate for at least six doctors, therapists and other new and strange faces.  Not a really comfortable position for Maya.

At the end of the appointment, the doctor asked the million dollar question. "If I call you and admit Maya, when could you be here?"  You can read my earlier posts to get a good understanding of how we answered the question.

Fast forward to today, and Maya's once experimental treatment has received FDA approval, our insurance company has agreed to pay for the treatment and we have transferred to our new hospital, Children's Hospital of Orange County (CHOC), only 90 minutes away from home.  Each step to this moment in time, required the efforts of so many people, many sacrifices, so many prayers and so much good luck, but we made it.

Yesterday, as we were wrapping up Maya's infusion, we sat and spoke with our friends Bekah, Danny and Ely (Maya and Ely have received infusions on the same day since last October and we transferred to CHOC together).  Bekah and I discussed the surreal feelings of the day.  So much worry, so much stress as our families traversed so many milestones and challenges during the last year.  Yet, here we were, wrapping up our infusions, headed home to sleep in our own beds having experienced and accomplished so much in such a short amount of time.

And so, for dinner last night, we went to Benihana for a post infusion celebration.  Not just a celebration for this day, but a thank you to those who helped make this day possible.



Although things have understandably slowed down with our #FightingForMaya efforts, we continue to work hard to advance this campaign.  Most important we continue to support the research project at Texas Children's Hospital and to try and find others with Maya's specific diagnosis.  Despite the fact that the Enzyme Replacement therapy is available, we need to continue to prime the research/treatment/cure pipeline.  From a purely scientific perspective, we know that the enzyme replacement therapy will significantly slow the disease's progression and there is a reasonable chance that Maya may regain some functionality.  However, we don't know what exactly to expect and the therapy is not a cure.

We also know that this treatment only addresses the brain, is very invasive and very expensive.  We know that down the line other major organs may be impacted and we will need to have several tested, viable treatments available to address future issues in other organs.  It is also important to take steps now to provide less expensive treatments so that everyone is able to receive treatment without delay.

Perhaps, more important, we need to make sure that a cure is available.

To all of our #FightingForMaya supporters, thank you for helping us achieve so much in such a short period of time.

In a little more than a year, we have:

-Raised over $120k of the $200k needed for the research project at Texas Children's Hospital;
-Raised over $15k from sales of #FightingForMaya shirts alone;
-Distributed over 1150 #FightingForMaya shirts; and
-Thousands of #FightingForMaya pictures and videos have been shared on social media.

The awareness surrounding this campaign has been tremendous.  In a little under a year, we have found 4 new families with Maya's form of the disorder.  We've also received word, that more researchers are interested in studying Maya's form of the disorder and advancing the overall understanding of the classic and atypical forms of the disorder.

Each of these accomplishments could have only happened with your support.  Thank you.


Maya is doing well.

Yesterday was huge for her.  For the first time, since last October, Maya was able to sleep in her own bed after an infusion.  For Maya, who doesn't sleep well at the hospital, last night was an important milestone.

Maya continues to work really hard with her walking, speech and strength.  She can still only walk with assistance and her speech is slurred, but we continue to see small improvements.

Maya, as usual, continues to be her amazing self.  Inspiring so many people to accomplish so much.

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