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  • About Gaucher disease
    What is Gaucher disease?
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    • Enzyme replacement therapy
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    • Chaperone therapy
    • Ambroxol hydrochloride
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Featured Articles
June 2, 2025 News by Marisa Wexler MS

Gaucher disease gene therapy FLT201 shows proof of concept

FLT201, a gene therapy for Gaucher disease type 1 currently in clinical testing, led to sustained increases in glucocerebrosidase (GCase) enzyme activity and reductions in fat molecules in animal models, indicating that it works as designed. The gene therapy provides instructions for the production of an optimized version…

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May 30, 2025 by Marion Glaser

A message to the Gaucher community: We are a miracle

“I am a miracle,” an 8-year-old boy stated matter-of-factly. “We are all miracles. None of us was supposed to have survived this.” He showed me a chapter written about him in a book about Gaucher disease and read the first page aloud. He was an excellent reader. I glanced…

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May 29, 2025 by Rivka Silver

My unexpected diagnosis with Gaucher disease type 1

When most people imagine being diagnosed with a disease, they picture a cold, sterile doctor’s office and a somber physician delivering bad news. But my story didn’t start like that. I was a healthy kid with no red flags, no strange symptoms, and no reason to think I might have…

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Click the link below to find all of the latest news related to Gaucher disease.

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

    Gaucher disease gene therapy FLT201 shows proof of concept

  2. new column banner
    May 30, 2025 by Marion Glaser

    A message to the Gaucher community: We are a miracle

  3. new column banner
    May 29, 2025 by Rivka Silver

    My unexpected diagnosis with Gaucher disease type 1

  4. May 23, 2025 by Marion Glaser

    Solving Violet’s puzzle: Our journey to a Gaucher disease diagnosis

  5. A graphic shows the words CLINICAL TRIALS framing images of a bar graph, a pie chart, and a pill bottle.
    May 19, 2025 News by Marisa Wexler MS

    Gaucher gene therapy FLT201 shows sustained clinical benefits

  6. A baby is shown lying on her back.
    May 12, 2025 News by Andrea Lobo

    Monitoring lyso-Gb1 may aid choice to begin ERT in Gaucher disease

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