“Compared to the natural course of Canavan disease, in which most children do not achieve developmental milestones beyond that of a 6-month old, the fact that some children are sitting ...

Mani Foroohar’s rating is based on the promising initial data from BridgeBio Pharma’s Phase 1/2 CANaspire trial, which evaluated BBP-812, an AAV9 gene therapy for Canavan disease. The trial ...

Educates at-risk populations about Canavan disease and other Jewish genetic diseases and the reproductive options available to carrier couples. Encourages carrier screening for these diseases whenever ...

(RTTNews) - BridgeBio Pharma, Inc. (BBIO) Thursday reported positive preliminary data from the CANaspire Phase 1/2 study of its gene therapy candidate, BBP-812 for the treatment of Canavan disease ...

an investigational intravenous adeno-associated virus serotype 9 gene therapy in development for the treatment of Canavan disease. Highlights of the results presented at ESGCT 2024 include ...

BridgeBio Pharma, Inc. (NASDAQ:BBIO), a biopharmaceutical company focusing on genetic diseases, has reported encouraging preliminary data from a clinical trial of its gene therapy candidate, BBP-812, ...

and Canavan disease, both of which result from mutations in enzymes in the sulphatide pathway. There are no cures for these diseases, which are typically treated with enzyme-replacement therapy ...

“Compared to the natural course of Canavan disease, in which most children do not achieve developmental milestones beyond that of a 6-month old, the fact that some children are sitting independently, ...