News

Enrollment complete in Phase 1b trial of ABX1100 for LOPD

A Phase 1b clinical trial testing substrate reduction therapy ABX1100 in late-onset Pompe disease (LOPD) has completed enrollment, developer Aro Biotherapeutics said. This part of the trial (NCT06109948) investigates the medication’s safety, tolerability, and pharmacological profile in adults with LOPD. ABX1100 is given by intravenous (into-the-vein) infusion.

Nexviazyme shows long-term benefits for LOPD patients in trial

Using Nexviazyme (avalglucosidase alfa) led to sustained improvements in lung function, walking ability, and other motor skills over nearly three years of treatment in people with late-onset Pompe disease (LOPD), according to newly shared results from a global clinical trial. LOPD patients who switched to Nexviazyme after one…

Benefits of Pombiliti + Opfolda last for years, new data show

For adults with late-onset Pompe disease (LOPD), Pombiliti + Opfolda (cipaglucosidase alfa/miglustat) appears to offer lasting benefits. New four-year data from a Phase 3 trial show the combination treatment helped sustain improvements in motor function and disease biomarkers in patients who had previously been treated with standard…

Digital tech may help detect motor impairment in asymptomatic LOPD

Wearable digital health technologies (DHTs) may help identify subtle walking and movement impairments in people with late-onset Pompe disease (LOPD) that are not evident in standard clinical evaluation, a recent study in Italy shows. These technologies “may have important implications for management, follow-up, and treatment decisions in clinical practice,”…

Blood test ID’s where antibodies bind to Pompe disease ERT

Locations where antibodies bind to enzyme replacement therapy (ERT) and potentially reduce its effectiveness in treating Pompe disease can now be identified using a drop of blood, a study reports. Knowing where anti-ERT antibodies bind may help find interventions to diminish the immune responses that generate them and also…

Orthodontic care benefits young girl with infantile Pompe disease

Orthodontic treatment successfully addressed speech and swallowing problems in a young girl with infantile-onset Pompe disease (IOPD), according to a case report from Italy. “The approach outlined in this paper serves as an illustrative example of orthodontic treatment for addressing speech and swallowing difficulties in patients with Pompe Disease,”…

Myozyme may help LOPD patients walk, breathe over long term

Long-term use of Myozyme (alglucosidase alfa), marketed as Lumizyme in the U.S., helped individuals with late-onset Pompe disease (LOPD) maintain their ability to walk and breathe, while remaining generally safe and well tolerated over 15 years, according to a study in Italy. While responses varied, fewer than expected…