A Major Step Forward for H-ABC Gene Therapy

We have some very exciting news to share. Researchers at UMass Chan Medical School have received a $275,000 award from the National Institute of Neurological Disorders and Stroke (NINDS) to continue moving H-ABC/TUBB4A gene therapy research forward.

This means a great deal to our Foundation because we have been part of this work from the beginning. We helped bring UMass Chan and Yale together, provided early funding to get the research started, and continued to support the program as it developed.

More recently, when the researchers faced a serious funding gap, the Foundation reached out directly to NINDS leadership to explain why this work was so important and why it needed to continue. The project was reconsidered and has now received federal funding.

For a disease as rare as H-ABC, progress like this takes all of us. It takes families willing to provide samples and data, donors who make early research possible, researchers who are willing to take on an ultra-rare disease, and advocacy to make sure promising work is not overlooked.

This award is a big step forward, and we are incredibly grateful to everyone who helped get the research to this point.

October 20, 2020 H-ABC Foundation partners with UMass Medical School and Yale to study gene therapy for H-ABC