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, at 7:30 EST. 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 and take the audience on a journey through the lens of awareness and advocacy, beginning with an overview of the rare genetic condition and its challenges. Dr. Ozlem Goker-Alpan, an expert in Gaucher disease and Founder and CEO of the Lysosomal & Rare Disorders Research & Treatment Center, features to raise awareness about this condition.

This special edition covers important topics ranging from exploring treatment options, such as enzyme replacement and oral therapies, to looking to the future of research into gene transfer therapies which could reduce the need for frequent interventions to improve quality of life.

The show introduces patient advocates Cyndi Frank and Aviva Rosenberg, Co-Founders of the Gaucher Community Alliance who share an overview of the Alliance, their own experiences and how their organization is supporting the patient community. Speaking about working with patient advocates, the medical community and the industry teams, Rosenberg shares: “it’s the combination of those three legs that allows for the patient community to grow and feel empowered.”

Joined by Jim Howley, Global Lead of Patient Advocacy and Engagement at Prevail Therapeutics, who shares his own story, experiences, and how Prevail are looking to partner with the community to gain insights: “it’s more about learning how we can partner so that we’re not doing one thing, they’re doing another thing. We do it together and we get the best advice possible”.

Finally, The Balancing Act introduces a story of strength: a person named Fan who was diagnosed with Gaucher disease at just two years old. Despite facing tough times, surgeries, and medical treatments, Fan’s story is about resilience and hope. It’s a story showing what’s possible with effective treatments and a supportive community.

The episode gives an informative and engaging overview showing the challenges which accompany Gaucher disease. However, the message is one of hope, highlighting how awareness and community support can make a significant difference.

To watch the episode in full, visit Behind the Mystery of Gaucher Disease.

For more information or to contact Prevail Therapeutics, please email:

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

Get involved, stay informed, and connect with the Gaucher Community Alliance.

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