Measuring levels of glucosylsphingosine (lyso-Gb1), a diagnostic biomarker of Gaucher disease, in children confirmed by newborn screening programs to have the disease may aid a decision about when to start enzyme replacement therapy (ERT). That’s according to a study in Italy that measured lyso-Gb1 levels monthly in three…
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Gaucher disease doesn’t seem to have a major impact on conception, pregnancy, and childbirth, according to a study of women in five European countries. While half the women of reproductive age had irregularities in their menstrual cycles, only a few had difficulties conceiving. In some cases, however,…
VPRIV (velaglucerase alfa), an enzyme replacement therapy (ERT) for Gaucher disease, can be safely administered by patients or caregivers without posing additional safety risks relative to infusions administered by a healthcare provider, a study showed. ERT self-administration was also safe in people with Fabry disease who received…
