2025 Impact Report: How the SDS Community Is Advancing Research Toward Therapies

With gratitude, Shwachman-Diamond Syndrome (SDS) Alliance is proud to share our 2025 Impact Report—a comprehensive overview of how patient voices, scientific collaboration, and strategic investment are accelerating progress toward meaningful therapies.

This is the latest issue of the SDS Alliance Blog! Welcome to timely updates on all things SDS, Science, and Advocacy. We bring you a digest of recent scientific publications, conferences, and other newsworthy content - all relevant to SDS - with links to more details and learning opportunities. Are you interested in anything specific? Did we miss something? Let us know. Email connect@SDSAlliance.org or message us on Facebook! This is all for you!

Rare disease progress does not happen by chance. It happens when patients, families, researchers, regulators, and funders work together with shared priorities and clear direction. In 2025, the SDS Alliance focused on building the foundation needed to move from understanding SDS to delivering treatments that extend and improve lives.

👉 Read the full Impact Report here: www.sdsalliance.org/impact

What You’ll Find in the 2025 SDS Alliance Impact Report

The 2025 Impact Report highlights the concrete steps taken this year to advance SDS research and therapy development, including:

• Patient voice at the center of drug development We convened the FDA-facing Patient-Focused Drug Development (PFDD) meeting, supported by a community survey and live polling, to ensure that regulatory and research decisions reflect what matters most to people living with SDS.
  

• Clear priorities for therapy development Across surveys, discussions, and lived experience, one concern emerged consistently: reducing the risk of leukemia and bone marrow failure. The report documents how this priority is shaping research strategy and endpoint development.
  

• Clinical trial readiness and research transparency Through initiatives such as SDS POPS and publicly accessible recordings, we are closing long-standing gaps in access to information about research stages, timelines, and pathways—from early discovery through IND-enabling work.
  

• Infrastructure that enables progress From patient registries (SDS-GPS) to research planning tools, the report details how SDS Alliance is building durable infrastructure that supports collaboration, data-driven decisions, and responsible research investment.
  

• A powerful documentary film: Until There’s a Cure Created to bring forward voices that could not be present at the PFDD meeting, the film captures the lived reality of SDS and has become a standalone resource for researchers, regulators, and funders seeking to understand what is at stake.

Why This Impact Report Matters

Families affected by SDS cannot fund cures alone—and they should not be expected to. Progress requires coordination, expertise, and partnership.

Community support plays a critical role not only through dollars, but by showing researchers, funders, and regulators that the SDS community is engaged, aligned, and ready to participate in research. The Impact Report explains how SDS Alliance uses that support responsibly—pairing it with major research grants, formal collaborations, and milestone-driven investments to maximize real-world impact.

Read the Report and Stay Engaged

Whether you are a patient, caregiver, researcher, clinician, partner, or supporter, the 2025 Impact Report offers a transparent look at where we are—and where we are going.

📘 Read the full 2025 SDS Alliance Impact Report: www.sdsalliance.org/impact

Together, we are building the path toward therapies—and ultimately cures—for Shwachman-Diamond Syndrome.

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