Proving the power of global collaboration, the Castleman Disease Collaborative Network, an initiative dedicated to accelerating research and treatment for Castleman disease, convened 42 experts from 10 different countries to establish the first-ever treatment guidelines for idiopathic Multicentric Castleman Disease, based on data from over 300 iMCD patients. This landmark study was pre-published in Blood, the top hematology journal in the world.
The Orphan Disease Center in the Perelman School of Medicine at the University of Pennsylvania has launched a global registry for CDKL5 Deficiency, a severe epilepsy disorder with infantile onset. It involves cyclin-dependent kinase-like 5 (CDKL5), a protein whose gene is located on the X chromosome. The purpose of the registry, which has united hundreds of patients since its launch earlier this year, is to enable international collaboration to better understand CDKL5 Deficiency and to accelerate and improve clinical studies focused on developing new therapies to combat CDKL5.