clinical trials

In 2018, Dwayne Wilson was struggling under the weight of a Pompe diagnosis. Today he is living life to the fullest. Learn how he adjusted his thinking and adapted his life to thrive with Pompe disease.

Clinical trials can help researchers develop new treatments for disorders such as Pompe disease, which currently has no cure and few options for treating symptoms. Through these trials, scientists gather evidence to support the potential regulatory approval of new and better therapies that may improve the quality of…

RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups with limited resources build their own disease registries. The site was developed by the National Center for Advancing Translational Sciences (NCATS), a division of the National Institutes of…

Amicus Therapeutics is planning to launch a pivotal trial to compare its investigational therapy AT-GAA (ATB200/AT221) to the current standard of care available for patients with Pompe disease. The new trial is expected to provide additional clinical data to support Accelerated Approval for AT-GAA by the U.S.

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…

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.

The biotech companies Shire and NanoMedSyn have launched a new research partnership to evaluate a potential treatment for lysosomal storage disorders including Pompe disease. NanoMedSyn has developed an enzyme replacement therapy using proprietary technology called AMFA that was shown in preclinical models to have promising biological activity.