WHO SHOULD ATTEND?
As awareness advocates we want the whole world to know what the rare disease community faces, but we realize this festival has a niche appeal. Perhaps the key audiences are the following:
Geneticists, Genetic Counselors, Researchers, Doctors, Nurses
With 7,000 rare diseases no Doctor can be expected to be familiar with all of them. And even if you are already up to date on the clinical science these films place that knowledge in the context of how families actually live while facing these disorders.
In many cases of rare disease early detection can make a huge difference in outcome.
Patient Families and Advocates
Often patients and their families feel quite isolated as they face life with a rare disease. These films not only educate us on other conditions which may or may not be similar to the one affected our loved ones, but they show how much all rare families have in common. Also breakthrough in one disorder can often translate into treatment for a different disorder. This makes it all the more important to connect with other patient populations and their advocates.
Gene Therapy and Bio Science industry professionals
Sometimes you just need a reminder of why you do what you do. These films put human faces on the work you may already be doing and might inspire you to new avenues of applying your research.
Here's one of our favorite stories of a researcher getting an “aha” moment from a rare patient's mom. He asked “If we could fix only one symptom for your son what would it be?” “I'd like him to be able to talk,” was her answer. He thought a while and said “You know in the lab mice we see muscle paralysis in the jaw. We might be able to prevent that.” The mom was over the moon at the possibility. A possibility that hadn't occurred to the researcher, after all he was focused on curing the syndrome not necessarily relieving symptoms.
Should young children attend?
They are certainly welcome. Our films rarely if ever use language or visuals that would be rated PG-13 or R, but almost all of our films address mortality and many of them discuss the premature deaths of children. If your children have already been exposed to that message and it does not overly upset them, bring them along.