clinical trials

Amicus Therapeutics is recruiting participants for the fourth group of its ongoing Phase 1/2 clinical trial evaluating the company’s investigative combination therapy AT-GAA. The trial (NCT02675465), called ATB200-02, will assess the safety, tolerability, and efficacy of AT-GAA in different groups of Pompe disease patients. The trial has treated…

Interim results a from Phase 1/2 clinical trial support the therapeutic benefits of investigational therapy combination ATB200/AT2221 in patients with Pompe disease. Participants in the study showed improvements in motor and respiratory function. The therapy also resulted in a durable decrease of muscle damage biomarkers. These results will be presented…

Duke Health researchers are planning to launch a Phase 1 trial to test the safety of a new investigational gene therapy they developed to treat patients with Pompe disease. They are currently screening (NCT03285126) adults with late-onset Pompe disease to select eligible participants for the upcoming trial.

Results of a Phase 4 clinical trial support the effectiveness of Lumizyme (4,000 liters recombinant human GAA) for 52 weeks in maintaining clinical stability in Pompe disease patients previously treated with the therapy’s 160-liter formulation, called Myozyme. The study, “Efficacy, safety profile, and immunogenicity of alglucosidase alfa…