Gene Therapy Approval!

On Friday, April 17th, Emma’s neurology team shared the incredible news that she has been approved for Zolgensma, her gene therapy!

This moment did not come easily. There were several steps along the way that had to fall into place. First, Emma’s antibody levels, which can affect eligibility for gene therapy, needed to come down. While she was hospitalized in March, those levels were too high. But when they retested in April, they had decreased enough to make her eligible.

Next came insurance approval. We were prepared for a difficult process, as her doctors warned us that denials are not uncommon and approvals can be delayed. But with extra advocacy, support behind the scenes and several prayers, our insurance company approved Zolgensma the very next day.

From there, her neurology team worked quickly through additional steps with the hospital and pharmaceutical company. Because of their efficiency, we are now scheduled for Emma’s gene therapy on Tuesday, April 21st.

This will be a one-time, one-hour outpatient infusion, followed by a few hours of observation. If all goes well, Emma will be able to come home the same day!

With that being said, gene therapy does not come without risks. There is a possibility of liver complications, so Emma will be closely monitored with weekly lab work. She will also be on a steroid to help her body accept the treatment, which means we will need to keep her in a tight bubble to minimize the risk of her getting sick.

We are overwhelmed by the love, support, and generosity you have all shown Emma and our family. Every prayer, message, and t-shirt purchased has touched us in ways we can’t fully put into words. Emma’s prayer army is strong and moving, and we truly believe we are witnessing God’s work in action. We are stepping into this next phase of treatment with hope anchored in trusting that God is writing a story of strength, healing, and victory for Emma.