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  • About Gaucher disease
    What is Gaucher disease?
    Types
    Causes
    Symptoms
    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
  • Living with
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    • The Gaucher Guide to Grace — Rivka Silver
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    • The three Gaucher types
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Featured Articles
August 13, 2025 Columns by Rivka Silver

Breastfeeding has been a beautiful challenge for me as a new mother

Whether a mother feeds her baby by breast, bottle, pumped milk, or formula, it’s all sacred. Feeding a baby is an incredible act of love, and every mama is a superstar for sustaining life in whatever way she can. That said, breastfeeding has been the biggest blessing of my journey…

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This illustration shows a person eating an apple, with their digestive system highlighted.
August 11, 2025 News by Lila Levinson, PhD

Boy with Gaucher disease develops complication despite ERT treatment

A 4-year-old boy with Gaucher disease developed enlarged lymph nodes and a serious digestive complication called protein-losing enteropathy (PLE), despite being on enzyme replacement therapy (ERT) since the first weeks of life, according to a recent case report. While his symptoms improved with corticosteroid treatment and dietary changes,…

In this crossword-style illustration, the words
August 4, 2025 News by Marisa Wexler MS

Sharp gearing up to move Gaucher disease treatment ‘901 to trial

Sharp Therapeutics is gearing up to ask the U.S. Food and Drug Administration (FDA) for permission to begin testing its Gaucher disease treatment candidate ‘901 in clinical trials. The developer, a preclinical-stage biotechnology company, announced that it’s engaged the contract research organization Rho to help it put together its…

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. Main banner for Rivka Silver's column
    August 13, 2025 Columns by Rivka Silver

    Breastfeeding has been a beautiful challenge for me as a new mother

  2. This illustration shows a person eating an apple, with their digestive system highlighted.
    August 11, 2025 News by Lila Levinson, PhD

    Boy with Gaucher disease develops complication despite ERT treatment

  3. In this crossword-style illustration, the words
    August 4, 2025 News by Marisa Wexler MS

    Sharp gearing up to move Gaucher disease treatment ‘901 to trial

  4. new column banner
    July 30, 2025 Columns by Rivka Silver

    As a mother, I have to take care of myself as well as my child

  5. This illustration shows a blood-filled dropper next to four vials of blood.
    July 28, 2025 News by Lila Levinson, PhD

    Cytokines in Gaucher disease tied to early-onset osteoporosis in women

  6. Mice explore laboratory equipment in this drawing.
    July 21, 2025 News by Margarida Maia

    mRNA treatment shows durable effects in Gaucher disease mice

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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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