Today was a Good Day

A Big Win For Maxwell

This week marked a major milestone in our fight against SLC6A1. Our dedicated team of scientists produced the gene therapy treatment and are actively treating mice. If all goes as hoped, the mice will show improvement and this data will go before the Federal Drug Administration to approve the therapy for human use.

I have been so nervous that this day would be delayed, that I wouldn’t allow myself to get excited. I was at Children’s Hospital with Maxwell’s developmental pediatrician when I received the e-mail, and when I told her, she burst into happy tears. Another physician came in to celebrate with us, and in that moment the gravity of the day hit me.

I burst into tears, too.

We have been laser focused in our march forward to create this treatment, and we haven’t stopped to breathe, let alone reflect. Today’s happy news was a welcome moment of validation, and a reminder that the hard work, endless fundraising, sleepless nights, and agonizing uncertainty aren’t for nothing.

Everything is coming together… this is happening!

Thank you all for fighting along with us and giving Maxwell so much love. We have a huge army of loving supporters, and we need every one of you!

We told Maxwell the big news tonight and you can see his reaction for yourself:)

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