Karen Utley was honored to participate in the National Organization of Rare Diseases’ NORD Living Rare Forum in Houston, Texas. Several researchers stopped at the table and talked with her. It was a wonderful event connecting rare diseases and offering education on advancing science! There were workshops on advocacy at the government level, as well as workshops on how to gain the interest of industry, and how to balance rare disease work with life.

It was wonderful to see the work being done and share stories. Also, It was an honor to see Jayne Gershkowitz accept a NORD Rare Impact Award on behalf of Amicus! many of you will remember Jayne from our family conference and recognize Amicus as a company interested in CDKL5 Deficiency Disorder. Amicus has been working on pre-clinical gene therapy for CDKL5 since 2016.

Jayne Gershkowitz of Amicus Therapeutics with Karen Utley, President of IFCR