Gene Therapy Showing Promise in Treating XLRP

  Michel Michaelides, MD, of UCL Institute of Ophthalmology; Moorfields Eye Hospital provides an overview of X-linked retinitis pigmentosa (XLRP), including its symptoms, common treatments, as well as a summary of the latest interim data from the MGT009 trial. ...

Rare Diseases Clinical Research Network

Rare Diseases Clinical Research Network (RDCRN) The Rare Diseases Clinical Research Network (RDCRN) is an NIH-funded research network of 20 active consortia or research groups. CheckRare is proud to be collaborating with the RDCRN to raise awareness of their programs....

Hypoparathyroidism Explained

  Thierry Abribat, PhD, chief executive officer of Amolyt Pharma provides an overview of hypoparathyroidism. Persons with hypoparathyroidism show abnormally low levels of calcium in the blood and high levels of phosphorus (hyperphosphatemia).  Hypocalcemia can...

What is Von Hippel-Lindau Disease?

  Eric Jonasch, MD, Professor, Department of Genitourinary Medical Oncology at MD Anderson Cancer Center, gives an overview of von Hippel-Lindau disease. As Dr. Jonasch explains, von Hippel-Lindau disease is a genetic disease characterized by the formation of...