NewsUNITERARE5 days ago
The PAN Foundation's financial assistance program for Fabry disease patients is currently closed and not accepting new applications. Fabry disease is a rare genetic condition where the body can't break down certain fatty substances, causing problems in the heart, kidneys, and nerves. If you need help paying for Fabry disease treatment, you'll need to check back later or explore other assistance programs.
WHY IT MATTERSIf you have Fabry disease and were counting on PAN Foundation's copay assistance or medication support, you'll need to find alternative financial resources immediately since this program is not currently accepting applications.
Clinical trialCLINICALTRIALSApr 14
Researchers tested a new gene therapy called ST-920 for Fabry disease in 36 patients. This treatment uses a modified virus to deliver instructions that help the body make an enzyme called alpha-galactosidase A, which people with Fabry disease don't produce enough of. The trial is now complete and tested whether different doses were safe and well-tolerated.
WHY IT MATTERSThis completed Phase 1/2 trial is the first human test of ST-920, meaning results could help determine if gene therapy can provide long-term relief for Fabry disease patients who currently require lifelong enzyme replacement infusions.
ResearchPUBMEDApr 3
Researchers reviewed studies from the past 10 years about how artificial intelligence and connected care technologies can help patients with rare diseases, especially lysosomal storage disorders (LSDs)—conditions where the body can't break down certain substances properly. The review found that while these digital health tools show promise for helping doctors diagnose and manage these complex diseases, there are still big gaps in the research and not enough real-world examples of these technologies being used in patient care.
WHY IT MATTERSIf you have a lysosomal storage disorder, this research could lead to faster diagnosis, better remote monitoring through connected devices, and AI tools that help your doctors catch problems earlier—potentially improving your long-term care and quality of life.
NewsUNITERAREApr 3
The PAN Foundation's financial assistance program for Fabry disease patients is currently closed and not accepting new applications. This program previously provided up to $12,000 per year to help eligible patients pay for treatment costs. The fund status changed on April 2, 2026, and patients interested in applying should check the PAN Foundation website for updates on when it might reopen.
WHY IT MATTERSFabry disease patients who rely on financial assistance to afford expensive enzyme replacement or substrate reduction therapies have lost access to this $12,000 annual benefit, making it harder to maintain continuous treatment.