late-onset Pompe disease

A Phase 2 clinical trial testing the experimental oral therapy S-606001 as an add-on treatment for adults with late-onset Pompe disease (LOPD) has begun enrolling patients, according to an announcement from the trial’s sponsor, Shionogi. “This is an important milestone for Shionogi, as we continue to expand our…

ABX1100, an experimental substrate reduction therapy for late-onset Pompe disease (LOPD), was well tolerated and seemed to work as designed in an early clinical trial involving nine adults with LOPD, new data show. Developer Aro Biotherapeutics noted that, in addition to meeting the study’s primary endpoints, or goals,…

At this year’s Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, researchers and clinicians are talking about the central role of patients, families, and caregivers in driving change for people living with neuromuscular diseases. Bionews, the parent company of this site, sat down with John F. Crowley, president and…

Measuring levels of glucose tetrasaccharide (Glc4) in urine may offer a simple, noninvasive way to identify people with late-onset Pompe disease (LOPD) who are at higher risk of worsening muscle decline, even while receiving enzyme replacement therapy (ERT). That’s according to a real-world analysis…

ABX1100, an experimental substrate reduction therapy being developed by Aro Biotherapeutics, showed strong and sustained biological activity and early signs of clinical benefit in people with late-onset Pompe disease (LOPD), according to preliminary trial results. The therapy was also well tolerated. The preliminary results are based on the…

Women with late-onset Pompe disease (LOPD) can safely use enzyme replacement therapy (ERT) during pregnancy and breastfeeding, according to a case series. Of the five reported pregnancies from three women, all were uneventful, and all five children breastfed by mothers receiving ERT exhibited normal growth and age-appropriate development.

The U.S. Food and Drug Administration (FDA) has cleared AskBio’s investigational new drug application for AB-1009, a potential one-time gene therapy being developed for late-onset Pompe disease (LOPD). The clearance enables the therapy to proceed to a Phase 1/2 clinical trial (NCT07282847)…

A viral vector engineered from a duck virus, called AAV.div3A, may offer a way to safely give patients more than one dose of gene therapyaccording to a study. This is something that is largely impossible today, yet critical for diseases requiring early treatment and that may need additional doses over…

For people with late-onset Pompe disease (LOPD) who are diagnosed before developing symptoms, treatment can be delayed until signs of the condition become apparent, but regular monitoring is essential so that therapy is not delayed when it is needed. That’s the argument made by a team of scientists in…

Late-onset Pompe disease (LOPD), a rare, inherited muscle disorder, was finally diagnosed in a 52-year-old man in Austria after decades of misdiagnosis, a case study shows. The condition had long been hidden by overlapping symptoms of dermatomyositis, an autoimmune disorder. Only after treatment for the autoimmune disease failed to…