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 ...

which are key biomarkers for Canavan disease due to ASPA gene mutation leading to NAA accumulation in the central nervous system. Despite the small commercial opportunity, with around 1,000 ...

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 ...

(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.

Affecting approximately 1,000 children in the U.S. and European Union, Canavan disease is an ultra-rare, disabling and fatal disease with no approved therapy. Most children are not able to meet ...