The newest addition to the NORD Rare Disease Video Library is a set of four videos on genome editing, a promising approach to treatment for many people affected by rare diseases. The videos address questions shared with NORD by our members and social media followers.
These short, animated videos answer… Read More
The results of a survey on gene therapy conducted by NORD and Frontline Medical Communications (FMC) among physicians and other healthcare professionals were recently announced. The responses indicated significant knowledge gaps among providers across 14 specialty areas.
For instance:
This webinar will feature individuals who have undergone gene therapy procedures. Don’t miss this exciting opportunity to hear people share their personal journeys with gene therapy. What were their symptoms before gene therapy? How did they find a clinical trial or a doctor? What were the necessary preparations… Read More
By Debbie Drell, Director of Membership
My sister was diagnosed with a rare disease, pulmonary hypertension, in September of 1998. At some point during her diagnosis, she was told she wouldn’t live to see her 30th birthday. She was 28. There was one… Read More
