Today, February 28th, is Rare Disease Day 2021. It is a day recognized around the world as a global campaign of awareness. What is a rare disease? A rare disease is any condition affecting less than 200,000 people within the United States. It is estimated that 10% of Americans are living with a rare disease. Multisystemic Smooth Muscle Dysfunction Syndrome (MSMDS) is an Ultra Rare disease affecting less than 50 children and young adults worldwide. Those affected with MSMDS are at high risk for stroke, aortic aneurysms, bladder dysfunction, respiratory disease, intestinal complications, and premature death. When your child is diagnosed with an ultra-rare disease, such as MSMDS, your life changes in many ways. You often find yourself consumed with finding answers; scouring the internet with hopes of finding guidance in treatment, or even another individual, that can relate to your journey. Suddenly, your time is spent managing doctor’s appointments, preparing for high-risk procedures, and clinging to hope that one day soon a treatment option may be available to help your child. Unfortunately, 90% of rare diseases are currently without an FDA-approved treatment and only 7.9% of money spent by pharmaceutical companies on developing and manufacturing drugs is dedicated to rare disease treatments.
Why donate to rare disease and MSMDS? To give parents, like us, HOPE! Many foundations, like ACTA2 Alliance, need your help to further our mission and accomplish our goals. We greatly appreciate any assistance you can provide in increasing awareness, supporting families and funding research for our children with MSMDS.