On May 6, 2024, we hosted a virtual Focus Group that brought together, for the first time, over 50 participants representing the APBD and other Glycogen Storage Disease Type IV (GSD IV) communities.
The participants included patients, family members, patient advocacy organization leaders, clinicians, and researchers from 13 countries.
All GSD IV disorders, including APBD, are caused by mutations on the GBE1 gene. This results in many shared interests among community members. A recently completed study by the Broad Institute of MIT and Harvard estimates the global genetic prevalence of all GBE1 diseases, including APBD and other GSD IVs, at 26,000. To advance therapeutic development, we believe it is imperative to build bridges between the APBD and broader GSD IV community.
Watch the recordings from researchers who spoke at the Focus Group
Read the Summary Report
We welcome the opportunity to strengthen and unite the APBD and GSD IV communities. It is through our strategic collaborations that we can gain momentum, build strength in numbers, accelerate research, and make treatments a reality.
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