We’re excited to share that the APBD Research Foundation is now a proud member of the Consortium for Outcome Measures and Biomarkers for Neurodevelopmental Disorders (COMBINEDBrain).
As an incoming member, we join over 92 patient advocacy groups working together to speed the path to clinical treatments for people with severe rare genetic, neurodevelopmental and neurodegenerative disorders by pooling efforts, studies, and data.
COMBINEDBrain was founded in 2019 by Dr. Terry Jo Bichell, who worked as a documentary filmmaker and then a public health nurse-midwife until her youngest child, Lou, was diagnosed with Angelman syndrome in 2000. In a quest to find treatment options for Lou, she dedicated the rest of her career to advancing research and clinical trial readiness for rare neurodevelopmental disorders.
Through this partnership, we have access to a wide array of impactful research initiatives, including:
- expanding diagnostic tools and access
- supporting development of cell and animal APBD models
- creating a patient-led biorepository
- advancing natural history studies
- collaborating on outcome measures and clinical trial endpoints
- navigating FDA pathways with greater support
- tapping into scientific and industry collaborations
- exploring opportunities for drug repurposing
A key focus of our involvement is launching a patient-led biorepository, a vital resource that will allow researchers and drug developers to access high-quality tissue samples from our community. This will help drive discovery and development of treatments for APBD and related conditions.
Jeff Levenson, DDS, co-president of the APBD Research Foundation, shared, “This partnership doesn’t just aim to develop treatments, it strengthens every step along the way, from diagnosis and research to trial readiness. By working synergistically, COMBINEDBrain allows its members to move faster and smarter together.”
Stay tuned for information in the coming weeks about how you can participate.