A desensitization protocol allowed doctors to successfully treat an infant with classic infantile-onset Pompe disease (IOPD) who developed an allergic reaction to Myozyme (alglucosidase alfa), according to a report. The protocol, which was also used when the patient switched to Nexviadyme (avalglucosidase alfa), involved delivering the Pompe…
enzyme replacement therapy
Children with infantile-onset Pompe disease (IOPD) experienced reductions in disease-related biomarkers and stabilizations or improvements in clinical function after switching from Myozyme (alglucosidase alfa) — sold as Lumizyme in the U.S. — to Nexviazyme (avalglucosidase alfa), according to a new study from Taiwan. These functional improvements were…
The combination therapy Pombiliti + Opfolda (cipaglucosidase alfa/miglustat) may help overcome some of the limitations of standard enzyme replacement therapies (ERT) for treating late-onset Pompe disease (LOPD), according to a recent presentation. Like other ERTs, Pombiliti + Opfolda contains a lab-made version of the enzyme that LOPD patients…
Higher-than-approved doses of enzyme replacement therapy, or ERT, normalized several features of infantile-onset Pompe disease (IOPD), including motor and heart function, in the first patient enrolled in a pilot study. The patient began high-dose ERT as a newborn, and eventually met all developmental milestones by age 7. With…
It’s no secret that the holiday season is a busy time for everyone, but imagine having to incorporate a 6.5-hour infusion therapy into the mix of it all. That’s the case for our family because my 6-year-old son, Cayden, has infantile-onset Pompe disease. While planning everything out can be…
Pombiliti plus Opfolda (cipaglucosidase alfa/miglustat) may offer significant advantages over earlier forms of enzyme replacement therapy (ERT) for Pompe disease, according to an analysis of preclinical and clinical data. Researchers reviewed all available data about the combination therapy’s mechanism and therapeutic effects and determined that it can overcome challenges…
Myozyme (alglucosidase alfa), sold as Lumizyme in the U.S., eased breathing but did not improve motor function of the arms and legs in an Iranian woman with late-onset Pompe disease who went undiagnosed for many years, according to a case report. The researchers say this case highlights the…
Recently, I read an article here at Pompe Disease News that brought me back to six years ago, when my son Cayden was a newborn. The article, “Twins do well on high-dose ERT plus immune tolerance induction,” reminded me of when Cayden was going through a similar experience. Cayden…
Late-onset Pompe disease (LOPD) patients who received severely under-dose treatment with enzyme replacement therapy (ERT) had a decline in physical quality of life, but an improvement in overall health, during a 14-month follow-up, a study in China shows. Being employed or enrolled in school predicted improved mental quality…
Early immune tolerance induction with rituximab, methotrexate, and intravenous (into-the-vein) immunoglobulin may have prevented twin babies with infantile-onset Pompe disease (IOPD) from developing antibodies against enzyme replacement therapy (ERT), according to a case report from the U.S. The report, “Optimizing clinical outcomes: The journey of twins with CRIM-negative…
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