Nexviazyme has worked wonders on my son’s lab results

It’s a relief to know all of the work it took to switch medications was worth it

Keara Engle avatar

by Keara Engle |

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It’s been a little more than 18 months since my 5-year-old son, Cayden, made the switch to Nexviazyme (avalglucosidase alfa) infusions as the main treatment for his infantile-onset Pompe disease. We started seeing improvements almost immediately, but now that he’s been on the new medication for some time, we’ve been able to watch his bloodwork levels continue to trend down.

Since his diagnosis when he was just a month old, Cayden has gotten routine lab work done. While it’s not enjoyable, it’s necessary so his doctors can see what’s going on inside his body. It’s easy to track and monitor physical symptoms, but it’s just as important to track the disease from the inside. 

Creatine kinase (CK) and liver enzyme levels are monitored closely in Pompe disease. The CK test is a very Pompe-specific test. CK levels are obtained from a urine sample and track muscle damage. The liver enzyme tests are necessary because Pompe disease can cause liver damage. A person with Pompe will typically have elevated levels of aspartate (AST) and alanine (ALT) aminotransferases, which are markers of liver damage that can be measured through a blood sample.

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Better numbers with Nexviazyme

From the first lab work results after making the switch to Nexviazyme until now, Cayden’s CK levels and liver enzyme levels have dropped significantly. At his diagnosis, his CK levels were 846. His most recent results were 851. (Normal range is 75-250). This means Nexviazyme has decreased his levels to the same range they were when he was a baby. While he was taking Lumizyme (alglucosidase alfa), the lowest his CK ever reached was 1,335 and the highest was 2,304. 

It’s such a big relief to know that all of the hard work it took to switch medications was worth it. Cayden has been getting enzyme replacement infusions since he was just a month old and newly diagnosed. He was initially started on Lumizyme as that was the only treatment available at the time. After a few years, we learned about Nexviazyme and that seemed to show more promise. Recently, Nexviazyme outperformed Lumizyme in the COMET trial, which is amazing news. 

I’m grateful there’s even one treatment option for Pompe disease, let alone more than one. The ability to make a choice is a blessing for those of us in the Pompe community. One medication may work well for one patient or another, or cause fewer side effects. Many rare diseases don’t have any treatment options at all, so I would say those of us in the Pompe community are blessed to have options. 

I hope one day a cure such as gene therapy is developed for Pompe disease. Until then, I’ll sit back and continue to be thankful for the Nexviazyme infusions that have been working so well for my son.

Note: Pompe Disease News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Pompe Disease News or its parent company, BioNews, and are intended to spark discussion about issues pertaining to Pompe disease.


Katty Rojas Osorio avatar

Katty Rojas Osorio

Es una gran noticia solo quería saber cuándo llegará este medicamento a Sur América ya que lo estoy esperando con ansias ya tiempo para mi menor hija ya que en Perú me an negado rotundamente en 3 ocasiones el aumento de medicina y veo que mi hija poco a poco va perdiendo fuerzas para caminar , necesitamos ese milagro lo más pronto para nuestros niños quisiera ya que se use también acá en Perú o darnos el beneficio de usarla en Mía lo más pronto posible


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