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How Shortages Affect the Rare Disease Community

It’s no secret that COVID-19 has caused a lot of issues for not just the United States, but the entire world. Because of the pandemic, for example, we’ve recently been experiencing issues getting the necessary medical supplies for my 4-year-old son, Cayden, who has infantile-onset Pompe disease. Cayden…

Antibodies to ERT Use Common in Children, Not Likely to Limit Efficacy

Most children with Pompe disease in a small study developed antibodies against the enzyme replacement therapy Myozyme (alglucosidase alfa) — marketed in the U.S. as Lumizyme — but their presence did not limit treatment efficacy, its researchers reported. Children with the highest levels of antibodies also experienced most of…

An Illness Doesn’t Have to Be Visible to Be Real

A rare disease is not always visible. Just because someone may look “fine” on the outside doesn’t mean that they don’t have struggles they face daily because of their disease. Pompe disease is one of those diseases. Pompe disease can onset any time throughout a person’s…

Welcoming New Pompe Parents to a Supportive Community Brings Me Joy

I will always be grateful I found our Pompe disease family. It brings me great joy when others find us and join the community, too.  A Facebook blog I made for my 3-year-old son, Cayden, started off fairly small. But over the…