August 11

Well, it's been a month.  A long and a difficult month.  When I wrote Little Buddy's update last month, I had no idea how much our lives would change by this entry.  Losing a clinical trial and hope will do that to you.  But still we are standing.  Still, we move forward.  

The second hardest part of this past month was watching Little Buddy get his last infusion (the hardest part was when we returned for his closing out blood work and the MPS conference and he DIDN'T get a treatment.  That nearly broke me, to be in the hospital and to not be able to get an infusion).  It hurt.  Every part of me hurt knowing that, unless something changes, his story is set.  Mimi was able to stay in town after she and Pop-Pop's visit so that she could stay with Little Sister while Daddy came with us to Minnesota.  I was very glad Daddy could come with us to this last treatment.

On our way to Minnesota.

Kind words and gifts from our hotel family.

     

His last port access.  His last infusion.  His last hope.  Any benefit is ENOUGH benefit when you have no treatment.  Even if this drug just helps him sleep and eliminates joint pain, that is enough.  I don't have the words to explain how much watching this broke my heart.

     

A wish in the fountain when we came home.  I think we all know what we were wishing for.

Even with everything we are feeling and experiencing right now, we are trying to keep things as "normal" as we can, and still trying to make summer memories.  

Painting time!

Bounce houses are both fun and good PT!

Justing chilling out and enjoying some soccer!

I somehow didn't get a picture of him swimming, but he did do survival swim lesson this summer.  This is him playing during Little Sweetie's lesson.  Prior to the trial his swimming was doing great!  He could be thrown into the water, hold his breath, come up, and swim to the other side, which is pretty amazing for a Sanfilippo kid.  Following the trial, he doesn't even want to be in the water.  He's so off and we hate that he doesn't enjoy the things he used to.

Height and Weight

Following the trial's cancellation, we have watched Little Buddy's energy and appetite diminish.  While he is 52.25 inches tall, he's dropped nearly 5 pounds and is now 61 pounds.  We're very concerned.  His appetite and weight was never something we had to worry about before.  

Personality

My happy excitable boy is missing.  Right now he is tired all of the time and, well, blank. Expressionless.  Showing no affect.  I don't know this little boy.  Did taking the drug away result in taking my son from me too?

Developmental Changes

We have experienced sudden and dramatic changes since Little Buddy was taken off of the clinical trial drug.  He won't feed himself, he'll barely even eat.  He can't stay awake and just falls asleep constantly.  He won't talk.  He will barely walk, let alone run, and he can no longer go up and down stairs by himself.  He can't even get himself out of bed.  We are so scared and heartbroken and working with our doctors to see if there is anything we can do.  If this keeps up, I don't know where he'll be in a month.  Things that were no problem a month ago are now tasks he cannot perform.  I expected quicker regression following the loss of the drug, but he's regressed nearly 3 years worth in just 3 week.  

Fun Tidbits

Here's the truth:  There's nothing fun right now.  We are all hurting.  We hate what is happening to Little Buddy.  But we have to focus on what we have and not what we are losing.  What we have are two kids that love each other and that need their parents to be strong right now.  And that is what we will work towards.

Super sibling love.

Hoping to report better news next month, 

LBM

Little Buddy's Momma

Our First MPS Conference!

It was a very bittersweet week for us.  With the trial ending, I had to take Little Buddy up to Minnesota for a few blood labs to be drawn to close out the study.  It was the first time in over two years that we wouldn't be there for an infusion.  Although the drug company doesn't believe that SBC-103 was helping my son "enough", we know it was.  Anything is enough when your child has a terminal disease with no treatment.  Any benefit is enough benefit.  And to be there and not be able to get the drug that was helping him...well, I can't put into words the heartbreak and hopelessness that I felt.

Last hospital trip.  I want to throw up.  

The only good thing about this week is the fact that the National MPS Society's annual family conference is also in Minnesota this week.  Blessed to be able to spend time with our extended Sanfilippo family, the only people who truly get the devastation that we currently feel.  And Daddy and Little Sweetie came up to join us for it to!  

The family is together again!

First stop on our Minnesota farewell tour, taking Daddy and Little Sweetie to Minnehaha Falls.  Little Buddy loves waterfalls and it's always a treat to see this one when we are in town.

     

Little Sister is so happy to be in Minnesota with her Big Brother!

The two boys resting up before going to check in at the conference.

The first thing I have to admit about the conference is that I didn't take nearly enough pictures!  Not even close.  Too busy meeting and talking to other families.  What a wonderful experience.

What great pictures of L, Little Buddy, and N!  So glad we got to meet these families.

The only thing better than a picture of these super Sanfilippo boys together?  Another picture including their super sisters with them!  

Worn out from the first day, but Momma is excited for all of the cuddles!  Ready for Day 2!

Cure Sanfilippo Foundation is representing!

The M family was there!  So glad to see them again! 

Little Sweetie was even more excited to see the M family and her buddy again!  They had such fun the entire conference!

     

These two are DONE!  

Day 3 consisted of a 5k race, a trip to the Sculpture Gardens, and a formal dinner.  Can't believe it was our last full day in Minneapolis.

Walking over the bridge to the race course.  I'm going to miss this place.

     

Second in my age group with my friend, D, right behind me!

Post-race family picture!

And then we FINALLY got to go to the Sculpture Garden!  Little Buddy has been obsessed with the picture of the spoon and cherry sculpture in our hotel for over 2 years.  We have wanted to go, but the  garden has been under renovation for nearly the entire time we have been in the trial.  Glad we got to check this item off our To Do list as a family.

     

The famous Spoon and Cherry!

     

Fun interactive sculpture!

     

A few of our favorites.

Glad that Little Buddy got to get out and walk around in the fresh air for a bit.  He's seemed very tired recently.  We'r hoping it's just the excitement of the conference and being off schedule.

Ready for the banquet!

     

More fun with the M family!

Sanfilippo Moms.  So glad to have these ladies in my life!

We're ridiculous when we're together.  And it's awesome.

     

Guess who does NOT want to leave Minneapolis?  You and me both, Little Sister.  Her face pretty much sums up my feelings about the end of the trial.

The last time we'll walk these hallways.

When you come somewhere regularly for 2 years, you make friends that become like family.  Our nurses, our rental car guys, our Chipotle workers, and especially our hotel staff.  So hard to say goodbye to them!  We were blessed by them and how they made us feel at home every visit.

And just when you think you are cried out, your cookie guy at the airport gives you a goodbye present.  We were truly blessed by everyone who loved on our family these past 2 years.

I'm still numb as I internalize the reality of this week.  While we were so glad that we could be with other families as we enter this new phase of Little Buddy's life, we are still just broken to know that he will no longer get any treatment.  We will research for other options and compassionate care for him but the reality is that things have changed and will never be the same again.  Little Buddy is dying and now he is receiving no treatment.  We have to find our new normal and focus on our family and our time together more than ever.  Because we don't know what the future holds or how much time we'll have together.

With sadness,

LBM

Little Buddy's Momma