by Peter Ciszewski | Jun 12, 2025
In 2007, the FDA created the Priority Review Voucher (PRV) program to incentivize the development of drugs for rare pediatric and tropical diseases. These PRVs act as a way to skip to the front of the FDA’s line to have a future drug reviewed, which offers...
by Peter Ciszewski | Jun 9, 2025
Stephanie Davis, MD, and Thomas Ferkol, MD, Professors of Medicine at University of North Carolina at Chapel Hill, and co-leaders of the Genetic Disorders Mucociliary Clearance Consortium, provide an overview of primary ciliary dyskinesia (PCD). PCD is...
by Peter Ciszewski | May 7, 2025
Progressive Familial Intrahepatic Cholestasis (PFIC): Diagnosing, Treating, Monitoring Patrick McKiernan, MD and Nadio Ovchinsky, MD This educational program, hosted by Patrick McKiernan, MD, Pediatric Hepatologist at Birmingham Children’s Hospital NHS...
by Peter Ciszewski | May 6, 2025
Consider Rare: Suspecting and Diagnosing Fibrodysplasia Ossificans Progressiva (FOP) Ellen Elias, MD and Christiaan Scott, MD Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic disorder characterized by abnormal bone development. Most babies with FOP...
by Peter Ciszewski | Jan 31, 2025
Improving Health Equity in Hereditary Angioedema (HAE): A Panel Discussion Aleena Banerji, MD, Timothy Craig, DO, Marc Riedl, MD Hereditary angioedema (HAE) is a rare genetic disease that results in immunologic attacks that can be life-threatening. HAE is the result...
