specifically the lysosomal storage diseases such as metachromatic leukodystrophy, and Canavan disease, both of which result from mutations in enzymes in the sulphatide pathway. There are no cures ...

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 ...

Eichler. “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 ...

(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, a rare genetic neurological disorder.

“We continue to be encouraged by the results children are achieving in the CANaspire trial of BBP-812,” said Dr. Eichler. “Compared to the natural course of Canavan disease, in ...

PALO ALTO, Calif. - 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 ...

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 ...