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
The potential for gene therapy products to change the lives of patients with debilitating or terminal conditions provides hope for the future. Gene therapy research is exploding at a rapid pace and a number of products are advancing in clinical development. The Center for Biologics and Evaluation Research (CBER) at… Read More
After decades of research and development, gene therapy is becoming a clinical reality in the US. With the first gene therapies already approved and many more in the pipeline, healthcare providers may soon gain the ability to treat more rare conditions.
To address this exciting topic, NORD is hosting a 5-part… Read More
