Research is critical for many, many rare diseases. Some people with rare diseases will not survive without treatments and interventions that can only be accomplished through research. Although specific treatments don’t yet exist for Moebius syndrome, there is a possibility that they could eventually exist and be used to help all of us. Plus the knowledge of the specifics of what all the causes of Moebius are would be welcomed by many people in the community. We’re lucky to have some very dedicated researchers interested in Moebius, definitely not an easy feat for an uncommon disorder!
Many other disorders aren’t as lucky, and have trouble securing the researchers and the funding to find potentially-lifesaving treatments. For Rare Disease Day 2012, NORD and Lundbeck are working together to raise funds for research:
Thank you for sharing your story in honor of Rare Disease Day. My aon Gage passed away from a rare genetic disease called early-onset LAL deficiency (also known as Wolman disease) in 2009. It is so important to be an advocate and speak up for our loved ones. My hope is that someday other families will not have to go through what we did, which is why I founded LALSolace — a support group for people and families affected by LAL deficiency. I wish your family well! Mary http://www.lalsolace.org