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  • About Gaucher disease
    What is Gaucher disease?
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  • Treatments
    Approved treatments
    • Enzyme replacement therapy
    • Cerezyme
    • Elelyso
    • VPRIV
    • Substrate reduction therapy
    • Cerdelga
    • Zavesca
    Experimental treatments
    • Chaperone therapy
    • Ambroxol hydrochloride
    • Gene therapy
    • FLT201
    • PR001
    Non-drug treatments
    • Orthopedic surgery
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    • Splenectomy
    • Stem cell transplant
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    • Growing Miss Violet — Marion Glaser
    • The Gaucher Guide to Grace — Rivka Silver
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Featured Articles
June 30, 2025 News by Margarida Maia

Gaucher disease diagnosis speeds up, but complications still common

The time from symptom onset to receiving a diagnosis of Gaucher disease has significantly decreased over the last decades, from a median of 5.4 years before 2000 to less than one year after 2020, a study in France has found. Still, researchers noted that, while most patients received standard…

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Main banner for Marion Glaser's column,
June 27, 2025 Columns by Marion Glaser

A week in the life of a working mom, wife, and caregiver

Last week was a busy one — so busy that I didn’t even have time to think about writing this column. I told my column manager as much. But as the week settled behind me, I realized it was full of moments — some good, some not — that are…

new column banner
June 25, 2025 Columns by Rivka Silver

Yes, I have Gaucher disease, but I don’t feel ‘sick’

As someone who is generally healthy and asymptomatic, I often feel like my Gaucher disease is tucked away in the back of my mind. Sometimes it creeps back in, reminding me, “Hey, I’m still here” — like when I get my infusion every other week or when my…

Main banner for Marion Glaser's column,

Featured ColumnOperation Sunshine Unicorn: A kayaking misadventure

The weather forecast was finally a good one, so columnist Marion Glaser signed her family up for “Operation Sunshine Unicorn.”

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  1. Two doctors confer while interacting with a tablet.
    June 30, 2025 News by Margarida Maia

    Gaucher disease diagnosis speeds up, but complications still common

  2. Main banner for Marion Glaser's column,
    June 27, 2025 Columns by Marion Glaser

    A week in the life of a working mom, wife, and caregiver

  3. new column banner
    June 25, 2025 Columns by Rivka Silver

    Yes, I have Gaucher disease, but I don’t feel ‘sick’

  4. A baby naps with her head on a pillow as a stuffed toy rests next to her.
    June 23, 2025 News by Steve Bryson PhD

    New Jersey’s newborn screening ID’s babies with Gaucher disease

  5. Main banner for Marion Glaser's column,
    June 20, 2025 Columns by Marion Glaser

    Operation Sunshine Unicorn: A kayaking misadventure

  6. new column banner
    June 18, 2025 Columns by Rivka Silver

    Motherhood allows no time for a break, even with Gaucher

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This site is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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