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…
Infantile-onset Pompe disease (IOPD) patients who develop antibodies against enzyme replacement therapy (ERT) exhibit a distinct immune profile from those who don’t, according to recent research. Patients who developed these so-called high and sustained antibody titers, or HSAT, were skewed toward a type 2 immune profile, which the…
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…
Switching from Lumizyme (alglucosidase alfa) to Nexviazyme (avalglucosidase alfa) was shown to improve breathing and motor function for most people with late-onset Pompe disease (LOPD) in a small real-world study. Many of the improvements, such as those seen in walking ability, were minimal, and did not reach…
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…
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