Switching to Nexviadyme (avalglucosidase alfa) from Myozyme (alglucosidase alfa) can help to halt the worsening of motor function in adults with late-onset Pompe disease (LOPD), according to a recent study in France. However, no significant differences in lung function were reported after patients switched these enzyme…
Most people with classic infantile-onset Pompe disease (IOPD) develop brain abnormalities despite treatment with Myozyme (alglucosidase alfa), and these abnormalities are associated with poorer results on cognitive tests. That’s according to a study, “Long term survival in patients with classic infantile Pompe disease reveals a…
Early treatment with at least two cycles of bortezomib may help lower the levels of antibodies that can make enzyme replacement therapy (ERT) less effective, according to a report of two young children with infantile-onset Pompe disease. The cases add to earlier reports that bortezomib, an injectable medication…
A powerful imaging technique called high-resolution intravital microscopy, or IVM, may be a useful tool for visualizing and quantifying the effectiveness of investigational treatments in mouse models of Pompe disease, according to recent research. The approach allows for cellular changes in muscles to be visualized in live animals. Using…
Sanofi’s Nexviazyme (avalglucosidase alfa), an enzyme replacement therapy approved for Pompe disease, doesn’t appear to be linked to new or worsening diabetes, a concern raised by the presence of dextrose, a simple sugar, in the solution for infusion. In a study funded by Sanofi, researchers also…
A gene therapy designed to travel into muscles resulted in widespread production of the enzyme that’s missing or faulty in Pompe disease, according to a study that also describes a new rat model of the infantile-onset form of the disease. Researchers also observed that rats given a single…
Using self-reported outcomes by adults with Pompe disease, researchers were able to estimate the minimal clinically important difference or MCID — the smallest outcome change that a patient perceives as important following treatment — after one year of enzyme replacement therapy (ERT). The differences reported by patients as clinically…
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…
Get regular updates to your inbox.