Stay Informed
Advocating for a Treatment
We Need You!
The Galactosemia Foundation is urging the U.S. Food and Drug Administration (FDA) to incorporate the experiences and perspectives of people living with galactosemia in the agency’s review of govorestat (AT-007), the potential first-ever treatment for our rare genetic disease.
Recently, Applied Therapeutics has issued a press release announcing while the ACTION-Galactosemia Kids trial data did not quite meet statistical significance, they believe it has demonstrated compelling evidence of clinical benefit and a favorable safety profile in patients with Galactosemia. They plan to request a meeting with the FDA to discuss a potential New Drug Application (NDA) submission.
Advocate Now!
We know advocacy can be time consuming and overwhelming, but it is more important than ever that our community rally behind this important cause.
It is going to take our entire community to help us get the attention of the FDA and let them know that we have waited long enough for treatment. There are many ways you can advocate, get started by using the following information below.
Our community has been busy advocating for a treatment. Here is a look back at some of the things we’ve accomplished.
Created a Petition
The galactosemia community urges FDA to incorporate Voice of the Patient in review of potential first treatment for patients with life-altering rare disease. Please spread the word by signing and sharing our Change.org petition.
Patients Rising Podcast
One mom’s journey into patient advocacy started after two of her daughters were diagnosed with galactosemia, a rare disease. There’s finally a potential new treatment for this condition, which families say has helped their children who have participated in the clinical trial. But they’re worried their voices are being ignored. Listen to the podcast here: https://www.patientsrisingpodcast.org/patient-voices-in-drug-research/
You can also share your story with a group of passionate passion advocates who believe the patient voice should be central to healthcare and barriers to access should be removed. https://patientsrisingstories.org/get-involved/your-patient-story/
Meet Amber
The Hill article
Share this article from The Hill written by our VP Brittany Cudzilo: The FDA is ignoring patients in clinical trials
Voice of the Patient Report
Read the Voice of the Patient Report which gives patient and caregiver perspectives on disease burden and unmet treatment needs in galactosemia.