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.
We know advocacy can be time consuming and overwhelming, but it is more important than ever that our community rally behind this important cause. Here are several ways you can advocate and tools to help you communicate your message and support our community.
But 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.
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.
Email us firstname.lastname@example.org: Share your experience with the clinical trial and/or your experiences with galactosemia and why you believe we need a treatment now. We will use quotes on social media. Please include a photo of your child/family if possible!
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/
Read the Voice of the Patient Report which gives patient and caregiver perspectives on disease burden and unmet treatment needs in galactosemia.
Share the report with family and friends and ask them to sign the change.org petition.
Share your story on your social media platforms. If you’re not sure what to say check out the examples below, or tell your story using the following question as a prompt:
What would a treatment for galactosemia mean for my family?” Use specific examples, such as: