GM2 gangliosidosis
IntraBio Inc. announced positive clinical trial results for IB1001, a pipeline drug intended to treat GM2 gangliosidosis. The multinational clinical trial met its primary endpoint, clinical impression of change in severity (CI-CS), as well as its secondary endpoints, which included patient clinical global impression of change (CGI-C) assessment, according to a press release from the company. IB1001 is the first drug to demonstrate a statistically significant and clinically meaningful effect for the treatment of GM2 gangliosidosis