Combining a symptom-based scoring tool with healthcare provider education may help identify patients who have an increased risk of having a missed diagnosis of late-onset Pompe disease (LOPD), a study in the U.S. shows. A LOPD diagnosis should be considered in patients with progressive muscle weakness and at least…

The U.S. Food and Drug Administration (FDA) has cleared Aro Biotherapeutics to conduct a Phase 1 clinical trial to test ABX1100, its experimental substrate reduction therapy, in people with late-onset Pompe disease (LOPD). The clearance of Aro’s investigational new drug (IND) application comes as the company is…

Late-onset Pompe disease (LOPD) patients who received severely under-dose treatment with enzyme replacement therapy (ERT) had a decline in physical quality of life, but an improvement in overall health, during a 14-month follow-up, a study in China shows. Being employed or enrolled in school predicted improved mental quality…

Treatment with Lumizyme (alglucosidase alfa) improved lung function in the short term and slowed lung function decline over the long term among people with late-onset Pompe disease (LOPD), according to a 13-year, real-world analysis. Patients with poor lung function before treatment saw the greatest early gains, while those…

Long-term treatment with Pombiliti plus Opfolda (cipaglucosidase alfa/miglustat) safely improved motor function and stabilized or enhanced lung function in adults with late-onset Pompe disease (LOPD), according to newly published data from two clinical trials. Pompe disease is marked by a deficiency in an enzyme called acid alpha-glucosidase…

An easy survey of 20 questions agrees well with other tools used in clinical practice to measure physical function in people with late-onset Pompe disease (LOPD), according to a study that suggests it may aid in measuring and tracking symptoms over time. Having a reliable way to measure physical…

Amicus Therapeutics has been honored with the WORLDSymposium 2024 New Treatment Award for Pombiliti + Opfolda (cipaglucosidase alfa/miglustat), the first medication duo to be approved for adults with late-onset Pompe disease. The award recognizes new treatments that received regulatory approval for lysosomal diseases like Pompe disease…

The use of a multidisciplinary treatment approach was described in a recent case report of a boy in the U.S. with late-onset Pompe disease (LOPD) and chronic pain. The strategy consisted of orthopedics, physical therapy, and psychosocial therapy. The boy also had behavioral and mood disorders, which could further…

The strength of hip muscles and gait wherein only one leg is in contact with the ground were identified as key parameters that affect walking and falls in people with late-onset Pompe disease (LOPD). Both could help assess the effectiveness of new therapies and should be “priority targets for…

Starting people with late-onset Pompe disease on enzyme replacement therapy (ERT) at any age can benefit their bone health, including reducing the risk of weak, brittle bones, or osteoporosis, a small study suggests. The study, “Effects of enzyme replacement therapy on bone density in late onset Pompe…