A newborn whose symptoms initially mimicked oxygen deprivation was ultimately diagnosed with Pompe disease, highlighting potential challenges in identifying the condition, according to a case report from Italy. This was an uncommon presentation, according to researchers, who described the child’s symptoms, which included an enlarged heart and weak…
News
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.
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…
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…
Switching from Lumizyme (alglucosidase alfa) to Nexviazyme (avalglucosidase alfa) stabilizes or improves lung function in people with late-onset Pompe disease (LOPD). That’s according to new analyses from the COMET Phase 3 clinical trial and its open-label extension study, which showed the benefits of switching to Nexviazyme were…
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,”…
Scientists at Sanofi have developed a new type of enzyme replacement therapy (ERT) that can clear excess glycogen — the underlying cause of Pompe disease — from the cells in the brain and spinal cord, which are untouched by current ERTs. That’s according to a mouse…
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 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,”…
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…
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