News

People with late-onset Pompe disease (LOPD) treated with Nexviazyme (avalglucosidase alfa) may be more than twice as likely to benefit from treatment than those on Lumizyme (alglucosidase alfa), a new analysis of data from the Phase 3 COMET trial suggests. The analysis employed a method called the…

Despite being on enzyme replacement therapy (ERT) for a long time, some children with infantile-onset Pompe disease (IOPD) can still develop problems in the brain and spinal cord, a small study suggests. Manifestations such as overactive bodily reflexes, encephalopathy (damage to the brain), and…

More late-onset Pompe disease (LOPD) patients treated with Nexviazyme (avalglucosidase alfa) achieved clinically meaningful improvements in patient-reported outcome (PRO) measures related to symptoms, daily life impacts, and life quality than those treated with Lumizyme (alglucosidase alfa). That is according to new analyses from the COMET Phase 3 clinical…

Children with classic infantile-onset Pompe disease (IOPD), who were first treated as infants with Myozyme (alglucosidase alfa) — sold as Lumizyme in the U.S. — continue to experience heart, breathing, and cognitive deficits, as well as motor problems, despite long-term use of the older therapy, a small German…

Throughout 2023, Pompe Disease News brought you coverage of the latest developments in research into Pompe disease and clinical trials of potential new treatments. Here is a list of the 10 most-read stories we published across that year. We hope to continue being a source of information…

The COVID-19 pandemic affected the treatment, as well as the physical and mental health, of people with Pompe disease, according to a recent survey of patients in 25 countries. More than 50% of respondents reported interruptions in enzyme replacement therapy (ERT) and physical therapy, affecting their disease, and…

Higher doses of Lumizyme (alglucosidase alfa) than what’s approved may improve survival outcomes for children with classic infantile onset Pompe disease (IOPD), according to a new study. An analysis of data from the Pompe Registry collected over nearly two decades showed that IOPD patients given Lumizyme at higher…

Pompe disease is less prevalent than expected in Spain, at around 3.1 cases per million inhabitants, according to a registry study that identified regional differences in prevalence, as well as the variable diagnosis and care workouts. “Our data supports the development of agreed guidelines to ensure that the care…

Mitochondria dysfunction is linked to hypertrophic cardiomyopathy — when the heart becomes enlarged and cannot pump blood efficiently — in people with Pompe disease, according to a new study from China. The disease-causing mechanisms were explored in a patient-derived heart cell model with characteristics of Pompe disease, including low…

A stem-cell based gene therapy that’s modified for better entry into muscle cells was seen to normalize protein levels in the skeletal muscle tissue of a mouse model of Pompe disease, according to a new report. The Pompe mice exhibited altered levels of hundreds of proteins — including ones…