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New Clinical Drug Trial for Galactosemia

Medical Disclaimer: This is my experience, and some of my emotions and what we do as a family for our Classic Galactosemia child. Always consult with your medical team about treatment and diet for Classic Galactosemia. The contents of this website, such as text, graphics, images and other material are intended for informational and educational purposes only and not for the purpose of rendering medical advice. The contents of this website are not intended to substitute for professional medical advice, diagnosis or treatment. Although we take efforts to keep the medical information on our website updated, we cannot guarantee that the information on our website reflects the most up-to-date research.Always discuss with your medical provider!

Not one of our team of Drs notified us of this study. We had to bring it up at our genetics appointment. SO bring it up with your Dr’s if interested. Honestly, I just went straight to Applied Therapeutics and got all my info.

Applied Therapeutics has a potential new drug AT-007 for people with Galactosemia. The preliminary results have been positive in adults. They are now looking to find pediatric participants 2-7.

Our family has decided to participate and should start in June 2020.(edit* since covid-19 things have been delayed a little but we are slowly sure surly going forward hopefully in August!) We simply emailed them(Victoria) and they got back to us quickly with answers to all our questions.

After covid settled down a little, a nurse came to the house to complete an assessment and to draw blood. She wore PPE and masks. It all sounds scary but these nurses who work for applied therapeutics and are professionals. The nurse even drove up in blacked out Escalade equipped with her own driver. Since my nurses arrival she has been our liaison. I can text call any time I want. we are waiting to fly out to their clinical Hospital in Atlanta to start the trial. They are even using private jets to keep us safe from covid-19. They are going above and beyond to keep us comfortable.

Many patients with galactosemia who follow a strict diet still end up with long term complications. Unfortunately the body still produces endogenous galactose. This drug may help that problem.

THEY STILL NEED PARTICIPANTS!!!!

If you are a patient or family member interested in receiving information regarding participation in the Phase 1/2 clinical trial, please  email: galactosemia@appliedtherapeutics.com

Click her for: Link to study information.

UPDATE: Covid-19 delays but today 6/24/2020 the nurse came out for blood draws and urine. This is the prescreening phase to see if we qualify. This is a controlled placebo trial which means we may get a placebo for study purposes. But we will be the first to get options of the real drug next phase. I’ll keep updating as we go through.

Update: 3/9/2021 we are still waiting to participate and should start May 2021.

If you have any questions about our experience please reach out!!