Find A Way

Those were the words of Merril Hoge, NFL great, ESPN commentator and cancer survivor.

I was of course skeptical (It's in my nature).  I've sat through many a corporate event where legends have described what has helped them achieve the impossible.

Listening to Mr. Hoge was a little different.  He humbly described himself as a "C" student of average speed, strength and height.  He humbly described how he indirectly received "the secret" from his idol, Walter Payton.... "I wanted it more.  I found a way".  It struck me that he described himself as an average guy who found a way to achieve the impossible.

Mr. Hoge described sitting on the couch, suffering through the excruciating pain of chemotherapy (perhaps feeling sorry for himself), when his young daughter climbed onto his lap and reminded him of the secret..... "Daddy, Want it more.  Find a Way".

Wednesday of last week, I traveled, with Ezra, to Pittsburgh for the 2017 BDSRA Batten Disease Family Conference.  It is a long trip to Pittsburgh and we decided that a red-eye to Newark and then Pittsburgh would be a lot for Maya.  Plus... truth be told... we didn't know what to expect.

My friend Alex nailed it on the head... this conference is not a "pity party".  It is a chance for friends, new and old, to greet each other with a hug and not have to "explain".  Yes, we enjoyed a weekend of not having to "explain".  Simply put, we celebrated this extended family.

It was a weekend where we met with researchers, clinicians, scientists and pharmaceutical companies, presented our children and reminded them that we want them to do better.  Yes, they have done much, but we want to "Find a Way".  Not just for our individual children, but our entire Batten Community.

It was a weekend where THE STRUGGLE was celebrated.

Personally, I'm reinvigorated (I've been exhausted).  I have my notebook of action items to close.  I have memories of long time friends, that I only this weekend met in person.  My heart is filled with new stories of children and families who are fighting the good fight.

I have memories of families who...

Want it More and are willing to Find a Way.

Road trip'n with Ezra.

Nothing like a custom Omelette.

Ezra's first "real steak".  Thank you, Ruth Chris, Pittsburgh.  In case you are wondering, the Steak won.

Our #FightingForMaya Post

Jacoby was the first person in the United States that I found with Maya's disorder.  This weekend was the first time that I met Jacoby and his family in person.

It was great to visit with Dr. Emily.  Our family's friend for life.

Shelli... a reminder that nurses make the world go round.

Heinz Stadium

Fort Pitt Park

Fort Pitt Park.  Before it was Fort Pitt, the French held this territory as Fort Duquesne.  The serendipity of this location and its history was not lost upon my deepest desire to better understand the root cause of Maya's disorder.

 Fort Pitt

Bridge over Fort Pitt.

The beautiful view from our room.

A little Football History.

Sunrise over Manhattan.

Sunrise over Manhattan.

Sunrise over Manhattan.

Sunrise over Manhattan. Upside of a red-eye into Newark.

Pioneer(s)

During our visit to Columbus for Maya's 5th infusion, Dr. Em sat on Maya's bed and in a very stoic tone, told Maya that she was a "Pioneer".  I cant help but think back to that day when I signed Maya's medical consent forms, I'm certain there was not a section called "Pioneer".  Six plus pages of risks and horribles, in very plain English, but nothing about Maya agreeing to be a "Pioneer".

Despite Dr. Em's proclamation, and there is some truth to it, I'd argue that there are so many other Pioneers that continue to step forward to battle Batten Disease.  At the risk of forgetting to thank others, I'd like to take a moment and thank the people of Columbus, Ohio for stepping forward in the battle against Batten Disease.

From the brave world class medical care at Nationwide Children's Hospital, to the unparalleled support of the employees of Nationwide Insurance, to sharing Maya's story, to embracing those who travel to Columbus for treatment, to bake sales, to providing us with a home away from home, to loving Maya and showering her with support, the people of Columbus have embraced our family, and many other Batten families, providing so many with unheralded love and support.  

For this, I am eternally thankful.

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You know, its kind of funny.  A friend recently told me that she and her friends were really looking forward to my next post.  I kind of looked at her and thought, "You and me both, sister."

Seriously, there is so much to say, and so few words.

I keep looking back at all of the pictures from the last 7 months and I just shake my head in amazement.  We've blown past our #FightingForMaya goal for year 1 and raised over $100k. In under 7 months, we have funded the first two years of the four year research project at Texas Children's Hospital.  THANK YOU!!!

My heartfelt dream is that soon, when a parent receives this devastating diagnosis, their doctor will write a prescription and say, "I've heard of this disorder.  Here is a course of treatment for your child."   Ambitious, perhaps naive, but possible.  Just ask my daughter, the "Pioneer".

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How is Maya?  Maya is a trooper.  She endures the pokes, injections, questions, new faces, old faces, security at the airport, boredom and so much more with a smile.

During the toughest moments, as she struggles with this disorder,  she will say "I don't want people to look at me".  

Its hard for her.  So very hard, but she is doing an amazing job.  We remain very hopeful that this treatment will stop the progression and restore some of the functionality that Maya has lost.  We remain vigilant.  

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Below are a couple of recent pictures and a wonderful video of support from Billy Zabka and Ralph Macchio.  Thank you Steve, Billy and Ralph.

As always, thank you for reading and for your love and support.

#FightingForMaya
www.fightingformaya.com

A #FightingForMaya Update

#FightingForMaya

When Maya was born, she had what we believe was an unrelated condition called gastroschisis, that required her to have surgery immediately after birth and spend five weeks in the NICU.  She was diagnosed during the 19th week of pregnancy and Suzette and I braced for her challenging, yet not impossible arrival.

I remember that her birth day was hectic. We had what we thought was a routine check-up with the obstetrician in the morning, however, we were told that Maya was entering into distress and would need to be delivered later that day. Because of her unrelated condition, Maya was delivered by c-section and was immediately whisked away to be evaluated by the pediatricians before her surgery. This part wasn't a surprise as Suzette and I had planned that I would accompany Maya to the surgery while Suzette went to post-op recovery.

As the doctors and nurses wheeled Maya to surgery, I realized that despite all of my planning, I hadn't planned for the reality that because of the c-section and need for immediate surgery, Maya had been born and Suzette hadn't seen her (Suzette heard her cry, but had not seen her).  A wave of panic hit me and I begged the doctors and nurses to not take Maya to surgery until I had returned with Suzette. I then took off running down the halls of the hospital, trying to find Suzette's room and someone to help me get her to Maya.

After much begging, I convinced a young intern to get Suzette out of bed, into a wheelchair and back to Maya. Luckily we found Maya, in an incubator and headed to the operating room.  Her surgeon, greeted us with a hearty smile and quickly agreed to give Suzette a few moments with Maya.  As I stood by the surgeon, and retold my tale of running around the hospital like a madman, he cracked a huge smile.  He then shared a bit of wisdom, and said, "There is nothing like the bond between a father and his daughter."

Little did I know.

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At some point, many of us parents in the rare disease community swear a little oath. You have that moment, perhaps after your child has regressed a little too far or perhaps when you question why your family has been tasked to bear such a burden. You silently swear to yourself, that no matter what happens to your child, that you will find some way to make it easier for those families that follow in your footsteps.  In some ways, I think that I made the oath, because no matter what happens (good or bad), I don't want any of this to be in vain.

Last month, after we returned to our hotel room in Columbus, recovering from the 48hrs in the hospital for Maya's first infusion, Suzette, Maya and I sprawled out on couches, beds and chairs.  We all just needed a couple of hours to recover.  I was staring at my phone, catching up on email and I came across a message titled "SCAR7 Diagnosis".  It was a note from a mom who had a 14 year old son who was diagnosed with SCAR7.  His geneticist had found our blog and passed along a printout and links.

I immediately gave the mom a call and during a thirty minute conversation, shared two and a half years worth of knowledge and understanding.  I then followed up with articles and introductions to Maya's doctors.

This young boy, represents our 21st person who is similar to Maya.  However, he is the first (and hopefully not the last) to have found us due to our #FightingForMaya work.

That moment and conversation meant a lot.


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As of this week:

- We have sold over 900 #FightingForMaya shirts;

-Raised over $13,000 from shirts alone;

-Over 600 people have either made a contribution directly to the SCAR7 research project at Texas Children's or bought a shirt;

-The FightingForMaya blog has been viewed over 18,000 times;

-We are six thousand dollars shy of hitting our year end goal of raising $100,000 of the $200,000 for the SCAR7 research project at Texas Children's Hospital.


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Our year end ask, is that you consider making a donation to the SCAR7 research project at Texas Children's.  Be this your first, second or tenth, we ask that you make another donation to help push us over the half way mark. This research project is so important in developing a targeted treatment for SCAR7 and expanding the understanding of this disorder.  We are close!!!

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Finally, I want you to know that Maya is doing well.  We are very fortunate to have her in the program at Nationwide Children's Hospital, Columbus and we make the trip every two weeks for her infusions.  Based on the published data, we can reasonably expect the progression to be slowed or stopped.  We hope and pray that over time, Maya will see some improvement.

As I have written many times, Maya (and our family have been showered with love and support). From tiny to mighty gestures, we have been so very fortunate.

In closing, we want to share a beautiful message that Maya received earlier this year (see below video).  It is one of many heartfelt messages (and we may share more), but we thought that we would share this one at this time. Thank you, Steve and Chris.

Thank you for reading and for your continued love and support,

#FightingForMaya
www.fightingformaya.com

Feeling Thankful

The first person I told was a dad I know.  His family is one of too many that have made the ultimate sacrifice to Batten Disease. I let him know that Maya received her first infusion today.  I shared with him how surreal it was to see the nurse walk into the room with the replacement enzyme that Maya needs.  He is a friend I look up to and respect.  I wanted to convey how thankful I was for his friendship, his family's sacrifice and their unparalleled dedication in working to save as many people as possible from the horror that is Batten Disease.

If you are confused, its okay.  You see, about a month ago, we were told that Maya had been accepted into an expanded use (compassionate use) program for an Enzyme Replacement Therapy.  I won't go too much into the therapy, but you can read about it here.  The results thus far have been very promising in terms of this therapy slowing the progression in kids with the classic form of the disorder.

In order to participate in this program Maya had to relocate to Columbus, Ohio for four weeks.  She will need to return every two weeks.  Suzette and I have commuted back and forth to be with Maya and to spend time with the boys back home.

Right now, I'm not up for writing much about the last couple of weeks  Except to say that I'm exhausted.  However, there are two important things that I want to get out there:

First,  the FDA is set to review this therapy for approval in early 2017.  I can't emphasize enough how important this review and approval will be for so many families.  Words can't capture it.

Second, I'm ridiculously thankful.  From my parents who jumped in at a moments notice to care for the boys during this last couple of weeks, to our friends who delivered meals, provided transportation, visited Maya, sent gifts, checked in on us constantly, worked untold magic in the background, to the insanely wonderful people of Columbus, Ohio who have shown us what makes the midwest so beautiful.  I can't even begin to explain how much people have opened their hearts and supported our family through this waypoint. (I will post more later about the great people of Columbus)

For now, that's about all I have to share.  Maya is good and in great spirits.  This is a monumental milestone for us, but we (i.e., #FightingForMaya) still have work to do.

Thank you for your continued love and support.