healthy volunteers

ABX1100 treatment for Pompe disease now in 1st trial in people

The first healthy volunteer has been enrolled in a Phase 1 study testing ABX1100 — a novel substrate reduction therapy from Aro Biotherapeutics — as a treatment for Pompe disease. This will be the first-in-human trial for the experimental therapy, meaning it’s the first time ABX1100 has been…

UK Trial Testing MRI Tool Seeks LOPD Patients, Healthy Volunteers

A small study in the U.K. is evaluating a noninvasive MRI-based tool that could potentially detect late-onset Pompe disease (LOPD) without the need for invasive muscle biopsies. This specialized muscle MRI also would monitor glycogen buildup — a hallmark of the condition — in muscles over time. The…