This Monday, many bridges, buildings, water falls, etc will be lit up to celebrate the innovation, courage, fortitude, and inspiration that embodies the rare disease community – from the strength of the patients and caregivers, to the empathy and compassion of health care workers, to the curiosity and resourcefulness of the researchers. Members of our rare community use Rare Disease Day to raise awareness about rare conditions with the rest of the populations. The day is also an opportunity to celebrate the incredible advances being made in how rare diseases are diagnosed, studied, and treated. And one of the leaders on that front is the National Institutes of Health (NIH). On Monday, the NIH will share some of the ways their researchers and collaborators are make game changing breakthroughs in basic and clinical science.
Please join virtually for Rare Disease Day at NIH next week on Monday, February 28 from 10 am to 6 pm to hear about new research advances, groundbreaking programs, patient stories/perspectives and more. Here is the link to the registration page: https://ncats.nih.gov/news/events/rdd
Highlights on the program will include:
- Insights into navigating the long and winding, often years-long road of the rare disease patient’s diagnostic odyssey, and a new NCATS programaimed at finding new strategies to shorten the path
- The latest in the development of personalized therapies for rare diseases
- Updates on NIH programs aimed at tacking more than one rare disease at a time, including the Platform Vector Gene Therapy project (PaVe-GT)and the Bespoke Gene Therapy Consortium (BGTC)
- How one small group of advocates and scientists drove advances in treatment and gave new hope for children and families battling a rare children’s autoimmune disorder
- Stories of overcoming obstacles during the pandemic, from forging new collaborations to recruit rare disease patients into clinical trials to creative ways for people with rare diseases to take advantage of telehealth
There’s always plenty to talk about. Please take a look at NCATS acting director Joni Rutter’s latest message to learn how NCATS is leading the way in rare diseases research, through programs such as the Rare Diseases Clinical Research Network and Therapeutics for Rare and Neglected Diseases, to breaking new ground in supporting advances in gene therapy and gene editing.
And don’t forget to wear your stripes.
