about the author:
Lauren Fritz has two sons with Duchenne Muscular Dystrophy. She is a private practice therapist and also cares for her sons fulltime.
Embracing “Both/And“
in the Duchenne Community
I find I am thinking a lot of both/and the morning after the FDA has granted the first ever accelerated approval for gene therapy for the treatment of Duchenne, in boys ages 4-5 only.
I understand the mechanics of this kind of approval. It is ground-breaking. It is the culmination of so much research and hardship. It is the start to other incredibly good things – both for our community and for other diseases. We can be happy and proud. This changes the trajectory of life for families with children who can benefit from this. Fundamentally.
And.
Stiff upper (quivering) lip. My job is to connect words around feelings. This does not take away from scientific progress.
Those families with children who are 6 years old or 9, or 12 or 15, or 20, 40… or deceased… there is deep sorrow. There is the feeling that “my individual child” – my wonderful son – is worth knowing and saving, too. His muscles are worth preserving. His life would have been worth preserving.
I know that when I look at the video of the sweet trial participants – who are young boys – it is with a combination of hope, wistfulness, and grief for my own children.
Both/And. We cannot ask our community to separate this. We do have the capacity to hold the joy and the grief together.
I am ecstatic for any family that does not have to take the route that we have taken, but I think we should speak to it all: the rejoicing and the loss. I know that in time, more boys will be helped by gene therapy. There is the honest awareness that this likely will provide limited help or access for our more progressed boys and young men. There is the wistfulness that this did not happen in ‘my time’ for so many.
I am rejoicing, and I am grieving. My grief is because my own children are important and dear, too. All of these boys and young men are important. All of those living, all of those who have passed… all of the families.
Many years of scientific innovation came together alongside the waiting and hoping and the disease progression yesterday. It is a strange journey. I know this is the work to get there. Yesterday was truly miraculous.
Both/And… Both/And.