Emma and Zolgensma

The following is a guest post by Emma’s Gramma Kathy.

Sometimes your heart speeds up a bit and you feel a surge of hope.

That happened  2 days ago when the FDA approved Zolgensma, the first gene therapy to treat SMA, for type 1 children.  Zolgensma delivers a healthy copy of the SMN1 gene that is missing. A video was published of a little boy that was part of the trial. He was running! AveXis  essentially saved his life.

This first approval was for the dosage given to babies up to age 2. Emma is 2 years 3 months and is not be eligible for this round.  There are more trials in progress for children over 2, that would include Emma. Assuming the trials have the same results, we could be looking at a whole new world.

So my heart speeds up… I feel so much hope for Emma to be free from sleeping with a bipap at night, from enduring the cough assist and suctioning machines, free of the pulse ox on her toe, free from the feeding tube in her little tummy. Do I even dare to hope that she could get into a sitting position by herself, or stand on her own, or walk?

Just to be not scared of losing her to a random cold would be enough.

Here is the link.  You may need a tissue.

Zolgensma Approval Press Release

NBC News Video – Zolgensma Results

Gramma