by Peter Ciszewski | Jul 16, 2024
In July of 2024, I visited Denmark and Italy to research their health systems as it pertains to rare diseases. In Denmark, I visited Copenhagen University and met with Oncology professionals to understand their views on rare diseases and rare blood disorders. I also...
by Peter Ciszewski | Jul 16, 2024
In July of 2024, I visited Denmark and Italy to research their health systems as it pertains to rare diseases. In Denmark, I visited Copenhagen University and met with Oncology professionals to understand their views on rare diseases and rare blood disorders. I also...
by Peter Ciszewski | Jun 9, 2024
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 20, 2024
Anne Pariser, MD, Director of the NCATS’ Office of Rare Diseases Research (ORDR), provides an overview of the ORDR and the research they are involved with. The ORDR is focused on multiple programs to improve the efficacy of rare disease research. Their two...
by Peter Ciszewski | Apr 5, 2024
The U.S. Food and Drug Administration (FDA) has awarded Rare Pediatric Disease Designation for SGT-003, a gene therapy designed for Duchenne Muscular Dystrophy (DMD) treatment. Duchenne Muscular Dystrophy (DMD) is a genetic disorder that leads to...
