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…
News
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,…
Children with infantile-onset Pompe disease (IOPD) who start enzyme replacement therapy (ERT) within the first month of life have healthier hearts than those who start the treatment later, a study found. “Our findings support the benefit of early ERT initiation in IOPD, particularly for cardiac outcomes,” the researchers…
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…
Avenue Therapeutics has secured rights to develop clenbuterol, an experimental add-on treatment for Pompe disease that has already shown promise in early clinical testing. The company announced it has entered into an exclusive worldwide license agreement with Duke University for patents and technical expertise related to the experimental…
A single dose of a specialized gene therapy has successfully normalized muscle function for more than a year in a mouse model of Pompe disease. By using “data-mined” genetic sequences designed to supercharge enzyme production specifically in muscle cells, researchers achieved protein levels up to 30 times higher than…
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…
A baby boy with infantile-onset Pompe disease and a severely enlarged heart was treated early with Nexviazyme (avalglucosidase alfa), a next-generation enzyme replacement therapy from Sanofi, leading to rapid and sustained improvement of heart function, according to a case report from Japan. “These findings may support 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.
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