Two girls in Colombia with early-onset Pompe disease saw their symptoms improve after methotrexate treatment reduced the levels of antibodies they had developed against enzyme replacement therapy (ERT), according to a case report. ERT antibodies can cause the therapy to stop working. Methotrexate, a chemotherapy, can work as an…
enzyme replacement therapy
In patients with Pompe disease, treatment with Myozyme (alglucosidase alfa) is most often administered in outpatient settings and is rarely associated with adverse reactions. That’s according to real-world data spanning a little more than seven years in France. The treatment is marketed as Lumizyme in the U.S. Overall,…
Every month, we receive a package at our house for my 6-year-old son, Cayden, worth almost $14,000. What could cost so much, you might wonder? Life-saving medication. Cayden receives biweekly infusions of Nexviazyme (avalglucosidase alfa), as enzyme-replacement infusions are currently the only treatment option for Pompe disease, which…
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…
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…
Note: This column describes the author’s son’s experiences with Nexviazyme and Lumizyme. Not everyone will have the same response to treatment. Consult your doctor before starting or stopping a therapy or changing its dose. My 6-year-old son, Cayden, has received infusions of enzyme replacement therapy for the entirety…
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…
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…
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