WE ARE ON A MISSION TO FIND A CURE
SMARD stands for Spinal Muscular Atrophy with Respiratory Distress (also known as SMARD1). There is little known about this ultra rare and often fatal disease. Its rarity is of no comfort for the families affected by SMARD, and so we are building a community to promote awareness and research to make a cure a reality.
Gene therapy research for SMARD is in progress, but we need YOUR help to raise approximately $1.5 million to keep the research moving forward. Gene therapy aims to replace or correct the faulty IGHMBP2 gene and is a well-documented treatment for this disease in animal models. The plan is to translate this promising data into human clinical trial as early as 2020!
This video shows success of gene therapy in mice at the Univesity of Milan (AAV-null mouse is untreated; AAV-IGHMBP2 has received gene therapy).
SMARD Family Spotlight
Meet Angel Kate
Click here to learn more about Kate's story.
I'm nine-month-old Landen from Kansas. Click here to learn more about me and my family.
Our small SMARD community is committed to spreading the word and doing all we can to get these wonderful kids treatment as soon as possible. For a critically ill individual, everyday is a celebration of life and we plan to celebrate each of these faces for many many more days and years to come.