Written by Laura Mullen 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 Laura Mullen 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 18, 2019
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
Written by Laura Mullen 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 Valaree DonFrancesco on November 20, 2019
During this webinar, clinicians who deliver potentially life-changing gene therapies will explain the gene therapy process and aftercare. Don’t miss this exciting opportunity to hear Dr. Jerry Mendell, Neurologist and Principal Investigator for the Center for Gene Therapy at Nationwide Children’s Hospital and Dr. Barry Byrne, Associate Chair of Pediatrics… Read More