Myozyme

Immunomodulators reduced anti-Myozyme antibodies, study shows

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…

Desensitization regimen can help manage anaphylaxis to Myozyme

Anaphylaxis, a severe allergic reaction, occurred in nearly one-third of children with Pompe disease treated with the enzyme replacement therapy Myozyme (alglucosidase alfa) in a small study. However, rapid desensitization regimens — using small doses initially and then increasing the doses — safely re-established tolerance to Myozyme in…

Myozyme Benefits LOPD Patient Misdiagnosed For 3 Decades

A woman was diagnosed with advanced late-onset Pompe disease after three decades of being misdiagnosed, according to a recent case report. After diagnosis, the woman began treatment with Myozyme (alglucosidase alfa), which led to marked improvements that enabled her to live independently. The findings underscore a need for…

Urinary Tract Problems May Be Common With IOPD, Despite ERT

Lower urinary tract symptoms, including a loss of bladder control and unusual urine flow, are prevalent among children with classic infantile-onset Pompe disease (IOPD) who begin treatment early with enzyme-replacement therapy (ERT), according to a small study. Findings suggest that “pediatricians should actively seek to recognize children with IOPD…