A Miracle for Ollie

11/18/2024

The anniversaries of the next few days are bittersweet. Today, 4 years ago, we were given the news that we were anxiously waiting for. Ollie did have MLD. I remember speaking with our neurologist on the phone in the parking lot of the high school. We were setting up an appointment to figure out our game plan for him, knowing ultimately that it would most likely involve moving to Italy so that he could undergo gene therapy. I prayed and hoped that it would be the miracle he needed to not have the same death sentence as Addi and so many other MLD children.
Now, today, I sit here watching him sleep, contemplating how different Ollie and Addi’s lives are. Gene therapy saved his life. He is doing things we only ever imagined for Addi. At 4, he is still walking, talking, can almost count to 20, knows 15 letters, can write his name, and does not suffer from seizures and respiratory failure. He is growing each day. Crushing goals and setting new ones. Nothing stops this sweet boy. He is literally a walking miracle. In this season of gratefulness there is so much to be thankful for. Ollie’s life, all of you for helping us get there, and that gene therapy is now an FDA approved therapy are just some of the reasons we are so thankful.

03/20/2024

This is so exciting! There are plans to have Lenmeldy available at 5 locations and one of those locations is Texas Children's Hospital!!

Five specialized treatment centers being qualified across the U.S. to minimize travel burden on eligible patients and their families Orchard Assist patient services program to provide individualized support throughout the treatment process Lenmeldy wholesale acquisition cost of $4.25 million for

03/20/2024

I had the honor of speaking to STAT News about the FDA’s approval of Lenmeldy, the gene therapy Oliver received and share our family’s story. How amazing is it that soon no other family will have to hear the words “There is nothing you can do, except keep her as comfortable as possible.”?

03/18/2024

MLD Gene Therapy was approved!!!! Jumping up and down over here!!🎉🎉

03/18/2024

Today is the day that we will hear from the FDA about whether the gene therapy Ollie received is approved for use in the U.S. To say I am on pins and needles and holding my breath is an understatement.

This therapy is absolutely life changing and will be a major step in the right direction. For too many children, like our Addi this was not an option as we found out too late. But this does not have to be the case soon!! With FDA approval and hopefully addition of MLD newborn screening to the RUSP, we will not have to see this disease steal anymore babies.

Praying and anxiously awaiting good news!

03/14/2024

03/11/2024

This is the gene therapy that saved Ollie! This is a huge step for the MLD Community.

The MLD Family™ and the entire MLD community is anxiously awaiting the FDA's decision on whether they will approve Orchard Therapeutic's MLD Gene Therapy for use in the United States. March 18th, 2024 is the day the FDA will announce their decision but it could be sooner. Stay tuned!

02/29/2024

Today is Rare Disease Day.

This is what Rare Disease means to us:

-It means choosing to live life to its fullest

-It means not focusing on the bad or the inevitable

-It means being followed by numerous specialists for both children

-It means countless doctor visits, and ER and hospital stays

-It means being able to recite your child’s medical history in a manner of minutes

-It means being an advocate

-It means special education, ARDs, and IEPs

-It means having PT, OT, and Speech

-It means we monitor Addis respiratory status constantly

-It means learning to use 9 different types of medical equipment to monitor and help Addi’s respiratory system

-It means doing around the clock respiratory treatments when Addi is sick and basically running our home like a miniature PICU

-It means we celebrate Addi’s ability to cough especially if it’s productive

-It means we celebrate when we don’t need to use those machines for Addi

-It means administering 28 different medications (including PRN or nebulizer medications) and constantly giving meds every 4 hours for Addi and 2 medications for Ollie

-It means knowing the signs of a seizure in your child and what to do for that seizure

-It means helplessly watching as Addi has a seizure

-It means watching your child have muscle spasms and neuro storms

-It means tube feedings for Addi and oral aversions for Ollie

-It means that the sink will always be cluttered with empty syringes and masks

-It meant Gene Therapy for Ollie

-It meant chemotherapy, bone marrow harvesting, and numerous blood infusions for Ollie

-It meant 6 months in Italy to save Ollie

-It means LOTS of sleepless nights

-It means trusting God through this journey

-It means praying for a cure

-It means praying constantly

-It means hopefully seeing newborn screening approval and FDA approved Gene therapy within their lifetime, but still too short to have saved Addi

-It means watching your other children grow into the most amazing and compassionate humans

-It means nurses that have become part of our family

-It means we celebrate that Addi can still talk to us through blinks and shows us her funny and sassy personality

-It means celebrating every new word or sentence Ollie learns

-It means celebrating every step Ollie takes

-It means we celebrate when Addi pushes hard and accomplishes the impossible like holding her head up

-It means celebrating Ollie’s “re-birthday” every year

-But more than anything it means we love, we laugh, we cherish each moment, and we FIGHT

02/14/2024