by CheckRare Staff | Sep 15, 2026
This week, three developments highlight the rapidly changing rare disease treatment landscape: a Phase 3 setback in Angelman syndrome, continued competition in hereditary angioedema, and an FDA decision expanding access to a targeted lung cancer therapy....
by CheckRare Staff | Sep 14, 2026
Fabry Disease: Patients’ Real-World Problems and How to Manage Them Staci Kallish, DO; Dawn Laney, MS, CGS, CCRC This educational program, hosted by Staci Kallish, DO, and Dawn Laney, MS, CGS, CCRC, examines how best to address patients’ real-life concerns...
by CheckRare Staff | Sep 9, 2026
In this episode of The CheckRare Brief, we discuss two recent FDA approvals for rare diseases, including Zanvastro for Alexander disease and Besremi for essential thrombocythemia (ET). We also examine a new gene therapy application for Huntington’s disease and...
by CheckRare Staff | Sep 9, 2026
For people living with rare endocrine disorders, getting an accurate diagnosis can be a long and frustrating journey. Symptoms may be subtle, overlap with more common conditions, or involve multiple organ systems, making it difficult for healthcare providers to...
by CheckRare Staff | Sep 3, 2026
In this episode of The CheckRare Brief, we discuss four recent FDA approvals, including LISRAYA for dermatomyositis and Imaavy for warm autoimmune hemolytic anemia (wAIHA), Mimrylo for polycythemia vera (PV), and Rasonque for pancreatic cancer. We also examine...
