Research Ready Data Collection Plan

We are excited to have created this GSD1b Patient Advocacy Organization to improve care, bring new therapies and treatments to GSD1b patients, and ultimately work towards developing a cure. Over the past 18 months we have worked closely with doctors, researchers, drug developers, policy makers and other rare disease organizations to get a better understanding of how we move the needle forward for GSD1b care and new treatments.

One of the most glaring areas of need, and one that we as an organization can control, is the need to be more organized and have a better understanding of our patient population. We have learned that providing researchers with our data is one of the most effective and efficient ways for us to accelerate our path to new treatments. Many folks have asked what they can do to help us move faster towards new therapies and a cure. This is your chance to help advance research for yourself or your child!

Please participate in the two very simple programs below. There is no cost to participate.

• Data Collection Program w/ RARE-X
• Patient Contact Registry

If you have any questions, please contact Jamas LaFreniere at jamas@sophieshopefoundation.org