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  • About Gaucher disease
    What is Gaucher disease?
    Types
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    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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    • The Gaucher Guide to Grace — Rivka Silver
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Featured Articles
August 27, 2025 Columns by Rivka Silver

Strength training with Pilates while living with Gaucher disease

Pilates workouts have become my “me time.” Once or twice a week, I step away from everything else — work, parenting, and the constant noise of daily life — and focus on myself and rebuilding my strength. After pregnancy, I felt especially weak, and Pilates offered a safe way…

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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

Treatment with corticosteroids and hydroxychloroquine to dampen inflammation helped ease serious lung problems in an infant with type 3 Gaucher disease, a case report shows. The child developed breathing difficulties and low blood oxygen despite being diagnosed early and starting enzyme replacement therapy (ERT). Because doctors suspected inflammation…

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?

Differences in nerve cell function may explain why some people with Gaucher disease develop the neurodegenerative Parkinson’s disease and others do not, a study found. When researchers compared nerve cells derived from siblings with Gaucher with different outcomes — one developed Parkinson’s while the other did not — they found…

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.”

Read the column

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

    Strength training with Pilates while living with Gaucher disease

  2. 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

  3. 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?

  4. 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

  5. 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

  6. 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

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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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