Written by Lisa Sencen on April 8, 2021
For several years, the National Organization for Rare Disorders (NORD) has prioritized gene therapy education to advance knowledge and promote safe and effective clinical applications to reduce suffering from rare conditions. With this goal in mind, NORD is dedicated to producing a variety of educational resources for patients, caregivers, health… Read More
Written by Valaree DonFrancesco on June 22, 2020
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
Written by Valaree DonFrancesco on April 16, 2020
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.
- 63% of respondents were unaware of current FDA-approved gene therapies.
- Respondents reported… Read More
Written by Valaree DonFrancesco on December 3, 2019
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
Written by Lisa Sencen on February 12, 2019
The following story was submitted by Lindsey McCarthy in honor of Rare Disease Day. In this story, Lindsey shares her family’s journey of receiving a diagnosis for their son, James, of a CNTNAP1 mutation. Read on to learn how the McCarthy’s have worked to find other families who… Read More