News

Recommendations issued on Pompe disease treatment switching

A group of experts in Europe published updated recommendations for late-onset Pompe disease (LOPD) treatment, including for the first time guidance about switching between different approved treatments. The recommendations were published in the European Journal of Neurology in a paper titled, “Start, switch and stop (triple-S)…

FORCE platform delivers ERT directly to muscles, nervous system

Dyne Therapeutics has unveiled promising preclinical data for its enzyme replacement therapy (ERT) FORCE-GAA — part of the company’s propriety FORCE platform — designed to treat Pompe disease. The novel therapy has demonstrated the potential to deliver the treatment directly to skeletal and heart muscles, as well…

Pompe children rate life quality better than parents: Study

Health-related quality of life for children with Pompe disease in the Netherlands is generally similar to healthy peers when self-reported, but worse when rated by their parents, a study found. Results also showed that children with classic infantile Pompe had greater fatigue and more limitations than those with non-classic…

Muscle MRI scans show promise for monitoring LOPD: Study

Quantitative MRI (qMRI) imaging of the muscles is a useful technique for identifying signs of compromised muscle health in people with neuromuscular conditions like Pompe disease, according to a small study. The study identified specific patterns of qMRI changes in the thigh muscles of people with late-onset Pompe…

Immunomodulators reduced anti-Myozyme antibodies, study shows

An immune system modulatory regimen effectively reduced levels of antibodies against Myozyme (alglucosidase alfa), an enzyme replacement therapy (ERT) for Pompe disease, in patients with low levels of acid alpha-glucosidase (GAA) enzyme. That’s according to data from a Phase 4 study (NCT02525172), which assessed such a regimen…

Phrenic nerve dysfunction tied to LOPD breathing issues in study

Dysfunction of the phrenic nerve, which is important for controlling breathing, was observed in a small study of late-onset Pompe disease (LOPD) patients who had ongoing respiratory dysfunction despite enzyme replacement therapy (ERT). “Our study provide new insights regarding respiratory mechanisms in LOPD,” the researchers wrote. Should their…