Nexviazyme

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…

Adding the enzyme stabilizer miglustat did not improve the effectiveness of Lumizyme (alglucosidase alfa) or Nexviazyme (avalglucosidase alfa) in a mouse model of Pompe disease, a new study reports. “These data demonstrate that co-administration of miglustat with either [Lumizyme or Nexviazyme] to Pompe mice provides no additional…

My 5-year-old son, Cayden, is no stranger to enzyme replacement infusions. He’s been getting them on either a weekly or biweekly basis since he was only 4 weeks old. It’s currently the only treatment option for Pompe disease, the rare genetic disease he was born with. Fortunately,…

Nearly three years of treatment with Nexviazyme (avalglucosidase alfa) led to long-term improvements in walking ability and lung function for people with late-onset Pompe disease (LOPD), according to new data from the Phase 3 clinical trial COMET. …

Infusions of enzyme replacement therapy (ERT) are something my 4-year-old son, Cayden, is used to at this point in his life. He started receiving them when he was just 1 month old, shortly after we got news of his diagnosis of infantile-onset Pompe disease through Pennsylvania’s newborn screening…

It all starts with the dreaded cough and snotty nose. Next thing you know, you’re rushing your child to the doctor to make sure they’re OK. These days, most parents don’t get too worried about a common cold, unless COVID-19 is a concern. But once that’s ruled out, things…

Recently, my 4-year-old son, Cayden, made the long-awaited change to Nexviazyme (avalglucosidase alfa) infusions, a new treatment option for Pompe disease. We have wanted to switch treatments for a while, but faced some insurance challenges that caused a delay. Nevertheless, we finally managed to get him switched, and…

The European Commission has approved the next-generation enzyme replacement therapy Nexviadyme (avalglucosidase alfa) to treat both late-onset and infantile-onset Pompe disease. This is the first time a new treatment for Pompe has been approved in Europe since 2006, according to Nexviadyme’s developer Sanofi Genzyme. “For more than…

Last week, we had our last home infusion for the summer. It was a bittersweet moment for sure. My 4-year-old son, Cayden, has been getting weekly Lumizyme (alglucosidase alfa) infusions from the same nurse at our home since he was about 18 months old. The infusions are necessary because…

A few weeks ago, I discussed how we were eyeing a medication switch to Nexviazyme (avalglucosidase alfa) for my 4-year-old son, Cayden. But switching to this new drug may not be as easy as his doctors and I were hoping. Cayden has been receiving Lumizyme (alglucosidase alfa) infusions…