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…
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…
Administrating a stem cell gene therapy before beginning enzyme-replacement therapy (ERT) prevented the development of antibodies against the acid alpha-glucosidase (GAA) enzyme, improved survival, and enhanced therapeutic benefits in a mouse model of Pompe disease, a recent study found. “Together, this study indicates that [stem cell] gene therapy induces…
Early treatment with enzyme replacement therapy (ERT) and immune tolerance induction (ITI) greatly improved clinical outcomes for infants with the severe, frequently ERT-nonresponsive, CRIM-negative infantile Pompe disease (IPD), a study has found. “Our data suggest that the first few weeks after birth…
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