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  • About Gaucher disease
    What is Gaucher disease?
    Types
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    Diagnosis
  • 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
    • Pain management
    • Physiotherapy
    • Splenectomy
    • Stem cell transplant
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    • The Gaucher Guide to Grace — Rivka Silver
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Featured Articles
September 15, 2025 News by Michela Luciano, PhD

Obesity may affect SRT treatment response in Gaucher disease

A 31-year-old woman with type 1 Gaucher disease (GD) experienced worsening symptoms and rising disease activity markers after switching from long-term enzyme replacement therapy (ERT) to Cerdelga (eliglustat), according to a recent case report. The woman — who was obese and had her spleen surgically removed years…

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Main banner for Rivka Silver's column
September 10, 2025 Columns by Rivka Silver

Reflecting on how I’ve grown stronger over the past year

As the Jewish new year approaches, I find myself in a season of reflection. I’m looking back on the past year and taking stock of how far I’ve come, while also looking ahead with hope and intention. Living with Gaucher disease means that I’m always aware of my body,…

A researcher looks into a microscope near a rack of vials and a beaker.
September 8, 2025 News by Marisa Wexler MS

Fatty molecule may cause nerve cell damage in Gaucher: Study

In Gaucher disease, the toxic buildup of a fatty molecule called glucosylsphingosine (lyso-Gb1) leads to issues with how nerve cells generate energy and other cellular problems, according to a new study. Lyso-Gb1 is a biomarker of disease burden and response to treatment. The findings help to understand how the…

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.
    September 15, 2025 News by Michela Luciano, PhD

    Obesity may affect SRT treatment response in Gaucher disease

  2. Main banner for Rivka Silver's column
    September 10, 2025 Columns by Rivka Silver

    Reflecting on how I’ve grown stronger over the past year

  3. A researcher looks into a microscope near a rack of vials and a beaker.
    September 8, 2025 News by Marisa Wexler MS

    Fatty molecule may cause nerve cell damage in Gaucher: Study

  4. Main banner for Rivka Silver's column
    August 27, 2025 Columns by Rivka Silver

    Strength training with Pilates while living with Gaucher disease

  5. A pair of red lungs connected to a windpipe.
    August 25, 2025 News by Lila Levinson, PhD

    Anti-inflammatory drugs treat lung issues in baby with Gaucher disease

  6. An illustration shows a pair of dice.
    August 18, 2025 News by Steve Bryson PhD

    Why do some Gaucher patients face Parkinson’s disease risk?

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Disclaimer

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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