by CheckRare Staff | Sep 29, 2026
This week on The CheckRare Brief, we are joined by Eugene Lee, CMI Media Group’s Group President, Specialty Communications, for an industry perspective on some of the biggest developments in rare disease. New Rare Disease Center of Excellence CMI Media Group recently...
by CheckRare Staff | Sep 24, 2026
KAT6 syndromes are rare genetic neurodevelopmental disorders caused by genetic mutations in the KAT6A or KAT6B genes. KAT6 syndromes are characterized by speech and motor delay, low muscle tone, feeding or gastrointestinal issues, sleep challenges, and vision issues...
by CheckRare Staff | Sep 23, 2026
My name is Dr. Joshua Zeidner, and I’m a Professor of Medicine and Chief of Leukemia Research at the University of North Carolina Lineberger Cancer Center. I’m here today to raise awareness around an active global, phase 3, double-blind trial evaluating a combination...
by CheckRare Staff | Sep 23, 2026
This week we discuss the FDA’s approval of Isembyld for SMA, the approval of a gene therapy for MPS IIIA, and the FDA’s Center for Devices and Radiological Health new Rare Disease Impact Initiative. Approval of Isembyld for Spinal Muscular Atrophy...
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....
