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  • About Gaucher disease
    What is Gaucher disease?
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  • Treatments
    Approved treatments
    • Enzyme replacement therapy
    • Cerezyme
    • Elelyso
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    • Substrate reduction therapy
    • Cerdelga
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    Experimental treatments
    • Chaperone therapy
    • Ambroxol hydrochloride
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    Non-drug treatments
    • Orthopedic surgery
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    • Splenectomy
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    Growing Miss Violet — Marion Glaser
    The Gaucher Guide to Grace — Rivka Silver
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Featured Articles
June 13, 2025 Columns by Marion Glaser

On teaching my children the benefits of volunteering

Last Saturday, my 7-year-old daughter, Violet, and I volunteered with Stream Watch, an award-winning program that takes care of high-use fishing sites on Alaska’s Kenai Peninsula. Stream Watch volunteers pick up litter, collect monofilament from recycling tubes, teach visitors about ethical angling, provide natural history education, and promote bear safety.

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An illustration shows a collection of pills, tablets, and prescription bottles scattered about.
June 9, 2025 News by Marisa Wexler MS

Casma selects treatment CSM-101 for Gaucher-related Parkinson’s

Casma Therapeutics has selected its first experimental therapy candidate, dubbed CSM-101, which will be developed to treat Gaucher’s disease patients who have Parkinson’s disease. The company is planning to submit an investigational new drug application to the U.S. Food and Drug Administration (FDA) in 2026 asking for permission…

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June 6, 2025 Columns by Marion Glaser

10 strategies that help us survive medical travel and appointments

Over the past year, our 7-year-old daughter, Violet, had 52 infusions and more than 22 additional medical appointments for her Gaucher disease. Because we live in rural Alaska, where there is no home healthcare, the infusions take place at our local hospital. For all other appointments, we travel —…

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Featured Column10 strategies that help us survive medical travel and appointments

To manage the stress and chaos of medical travel for her young daughter, columnist Marion Glaser prioritizes fun, family, and friends.

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  1. new column banner
    June 13, 2025 Columns by Marion Glaser

    On teaching my children the benefits of volunteering

  2. An illustration shows a collection of pills, tablets, and prescription bottles scattered about.
    June 9, 2025 News by Marisa Wexler MS

    Casma selects treatment CSM-101 for Gaucher-related Parkinson’s

  3. new column banner
    June 6, 2025 Columns by Marion Glaser

    10 strategies that help us survive medical travel and appointments

  4. Mice explore laboratory equipment in this drawing.
    June 2, 2025 News by Marisa Wexler MS

    Gaucher disease gene therapy FLT201 shows proof of concept

  5. new column banner
    May 30, 2025 Columns by Marion Glaser

    A message to the Gaucher community: We are a miracle

  6. new column banner
    May 29, 2025 Columns by Rivka Silver

    My unexpected diagnosis with Gaucher disease type 1

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