FBXO31 Foundation is excited to announce that two patients in our community have been accepted to pursue ASO treatment through n-Lorem.
If n-Lorem is successful these treatments will be individualized to each patient and will provide valuable information to the community on disease response to a targeted genetic therapy. n-Lorem is focused on creating individual treatments for patients with nano-rare diseases caused by a single gene mutation that affects less than 30 patients with the same mutation worldwide.
For Interested Patients
Presubmission Review
A good place to start.
An informal review of the mutation to determine if it might be amenable to n-lorem’s technology. If it seems like a good fit, a research physician would then need to submit a formal application.
Submit an Application
The next step.
Is your physician at an institution committed to your treatment if n-lorem is successful? Great! Formal applications can be submitted by a research physician through the n-lorem website.
Helpful Resources
n-lorem podcast series
Patient Empowerment Program
Creating a Community of Care
A forum where the voices of patients, advocates and experts can come together focused on the nano-rare patient