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Behind the Mystery of Gaucher Disease

Prevail Therapeutics features on Behind the Mystery, a recurring series on The Balancing Act devoted to advocating for rare and genetic diseases: Behind the Mystery of Gaucher Disease

The Behind the Mystery series clarifies and explains complex issues surrounding rare diseases. Featuring experts and key opinion leaders, the series is developed through partnerships with foundations and pharmaceutical companies and aims to ”amplify the voices of patients, families, and advocates affected by rare diseases.”

The episode, “Behind the Mystery of Gaucher Disease,” was launched on October 02, 2023. This ground-breaking episode unites experts from patient advocacy, healthcare, and industry, alongside someone living with Gaucher disease who has an inspiring story, offering hope to the patient community.

The episode aims to demystify Gaucher disease, a rare genetic puzzle filled with challenges, and take the audience on a journey. Dr. Ozlem Goker-Alpan, a renowned Gaucher disease expert, will guide this journey, highlighting the importance of early genetic testing and rapid intervention while exploring individualized treatment approaches alongside current and future therapies, including gene transfer therapies that could reduce the need for frequent interventions to improve quality of life.

We meet the patient advocates Cyndi Frank and Aviva Rosenberg, Co-Founders of the Gaucher Community Alliance, who have raised awareness and built strong bridges between the patient community, medical professionals, and the pharmaceutical industry. Alongside them, Jim Howley, an advocate from Prevail Therapeutics, reveals his inspirational path, emphasizing the transformative influence of patient advocacy in today’s collaborative medical arena.

Finally, we meet Fan, who was diagnosed with Gaucher disease at the age of two. Her remarkable story is one of resilience, hope, and the benefits that emerge when effective treatments and a supportive community stand together.

Watch the video in full.

For more information, please contact us:

Discover Prevail Therapeutics’ clinical trials on gene therapy for Gaucher disease and explore potential treatment options at or

Connect with the Gaucher Community Alliance for valuable resources and support at

To find out more about Behind the Mystery or The Balancing Act, please visit their website


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