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Illustration of corrected DNA symbolizing the edited SBDS gene for SDS with a question mark arrow pointing toward a patient figure, representing the promise and open questions of how to get gene therapy for Shwachman-Diamond Syndrome to clinical trials and into patients

How YOU can help move gene therapy forward for SDS

Gene therapy for SDS is advancing. Learn what's being developed, why base and prime editing matter, and how your perspective can shape what comes next.

SDS Alliance News

Eszter Hars, PhD

4 days ago

Hands holding a spiral-bound copy of the Shwachman-Diamond Syndrome Voice of the Patient Report, from the Externally-Led Patient Focused Drug Development Meeting held in Cincinnati, OH on June 4th, 2025.

One Year Later: The SDS PFDD Voice of the Patient Report Is Published — and Your Voice Is Already at Work

The Shwachman-Diamond Syndrome Voice of the Patient report is now published and submitted to the FDA. Learn what the EL-PFDD meeting captured, how the report is used, and what comes next.

SDS Spotlight

Eszter Hars, PhD

Jun 4

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How YOU can help move gene therapy forward for SDS

One Year Later: The SDS PFDD Voice of the Patient Report Is Published — and Your Voice Is Already at Work

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