Jul. 19, 2018
NORD’s RareEDU™ Launches Video Addressing a Topic Vital to Today’s Rare Disease Community, Gene Therapy: Your Questions Answered
Posted by Christina Jensen
Washington, D.C., July 19, 2018—The National Organization for Rare Disorders (NORD) today launched Gene Therapy: Your Questions Answered, a new video from its RareEDU educational program addressing some of the most commonly asked questions from patients and caregivers on the topic. It is available for viewing here.
“For 35 years, providing information and education to the rare disease community has been an integral part of our mission at NORD. Through RareEDU, we provide educational resources for patients and caregivers, medical professionals, students preparing for health-related careers and the public,” said Mary Dunkle, Vice President of Educational Initiatives for NORD.
“In light of the first therapies approved by FDA recently, gene therapy is a topic very much at the forefront of rare disease conversations. We are pleased to be presenting a video that patients, caregivers and the general public should find useful,” Dunkle added.
The goal of this video is to address the questions, hopes and concerns that patients and caregivers, across many different diseases, have about gene therapy. Since more than 80% of rare diseases are believed to be genetic, this video will serve as a helpful resource for the rare disease community.
Gathering input for the video was an interdepartmental effort earlier in 2018, with NORD’s membership team querying member organizations and the communications team requesting input from NORD’s social media followers. Though the responses received were from across the spectrum of rare diseases, the most frequently asked questions tended to be the same.
The responses were pared down to 11 of the most frequently asked questions. NORD then engaged with a social worker, Emma Crowley, and a medical expert, Dr. Cristina Liberati at the gene therapy center at the University of Florida medical school, to craft accurate answers to those questions. Marsha Lanes, a genetic counselor on the NORD staff, helped ensure that the responses were written in patient-friendly language and that video images were appropriate.
The video was made possible by a donation from Spark Therapeutics. NORD is solely responsible for the content.
Questions covered in Gene Therapy: Your Questions Answered include:
- Is gene therapy a cure?
- Why is gene therapy getting so much attention?
- Why does it cost so much?
- Will insurance pay for it?
- What is the difference between gene therapy and gene editing?
NORD will be developing other educational videos over the next several months on topics of interest to rare disease patients and caregivers.
About 7,000 rare diseases have been identified, affecting 25 to 30 million Americans. Of the 7,000 that exist, fewer than 500 rare diseases have FDA-approved treatments.
About the National Organization for Rare Disorders (NORD)®
The National Organization for Rare Disorders (NORD)® is the leading independent advocacy organization representing all patients and families affected by rare diseases. NORD is committed to the identification, treatment and cure of the 7,000 rare diseases that affect 30 million Americans, or 1 in every 10 people. NORD began as a small group of patient advocates that formed a coalition to unify and mobilize support to pass the Orphan Drug Act of 1983. For more than 35 years, NORD has led the way in voicing the needs of the rare disease community, driving supportive policies and education, advancing medical research, and providing patient and family services for those who need them most. NORD represents more than 270 disease-specific member organizations and their communities and collaborates with many other organizations in specific causes of importance to the rare disease patient community.