Azrael's Wish

01/22/2022

Really hoping and praying the FDA listens to us parents with children suffering from Galactosemia - a rare and devastating disease. We need this treatment to get FDA approval

As many of you know, my eldest daughter with Classic Galactosemia is currently enrolled in a clinical trial that is researching the first ever possible treatment for Galactosemia. To put this into perspective, only 5% o...

09/15/2021

The toughest kid - she’s in Michigan participating in day 90/91 of the applied therapeutics at007 clinical trial

06/16/2021

Prayers for Azrael today. It’s dosing day She will either get the actual AT-007 drug or the placebo. We won’t know as it’s a double blinded clinical trial

She will have blood draws and vitals throughout the day - we are here for 14 hours

We are both nervous and hopeful this drug could be a life changer for her and others like her I’m amazed at her bravery and strength throughout all this and everything life has handed her over the years

06/14/2021

Super brave Azrael is in Michigan participating in clinical trials for a possible drug treatment for Galactosemia. So proud of her and all the other strong kids for their bravery
-007

06/12/2021

https://www.appliedtherapeutics.com/patients-caregivers/understanding-galactosemia/

Galactosemia is a rare, slowly progressing metabolic disease caused by a genetic inability to break down the sugar galactose. Learn if you're at risk.

03/31/2021

This little miracle is 13 today. Happy birthday Azrael

12/09/2020

Applied therapeutics just launched this website

Learn about Galactosemia, including information on what this rare disease is, what causes it, who's affected by it, the types of Galactosemia, & more.

09/17/2020

The best video I’ve have seen explaining galactosemia in a nutshell

This is "Galactosemia: A Tale of Two Pathways" by Applied Therapeutics on Vimeo, the home for high quality videos and the people who love them.

09/16/2020

09/03/2020