We are on track to complete two important milestones in the first quarter of 2025-first, agreement with the FDA on the design of a PSC pivotal trial for CM-101 and second, reporting new clinical data ...

The study is being conducted under a Special Protocol Agreement (SPA) with the U.S. Food and Drug Administration (FDA).

Their results argue for a change to parity between small-molecule agents and biologics when it comes to Inflation Reduction ...

Many seriously ill people die waiting for the FDA to approve drugs that regulators in other advanced countries have already ...

In a groundbreaking study, researchers at McMaster University have identified a potential treatment for Sandhoff and Tay-Sachs diseases—two rare, often fatal lysosomal storage disorders that cause ...