Most of the time, I forget I even have Gaucher disease. I don’t wake up feeling sick. I don’t move through my day with pain or obvious limitations. I work, take care of my family, make plans, and lead a full and busy life. If you passed me on…
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Measuring the amount of glucosylsphingosine, also called lyso-Gb1, a key marker used to help diagnose Gaucher disease, may aid doctors in deciding when someone with Gaucher disease type 1 should begin treatment, a new study suggests. Additionally, a certain level of lyso-GL1 may help distinguish between patients who are…
VPRIV (velaglucerase alfa), an approved enzyme replacement therapy (ERT) for Gaucher disease, appears to be safe and effective in children younger than 4 — an age group not included in the clinical trials that supported the treatment’s approval. That’s according to a new U.S. real-world study that…
