Shwachman-Diamond Syndrome Alliance

08/08/2026

🎉 Big news for the SDS community — SDS POPS is coming to Paris in 2027, in person for the first time!

In May 2027, adjacent to the 12th International Scientific Congress on Shwachman-Diamond Syndrome, SDS Alliance is planning a community gathering in Paris:
* an informal dinner on Saturday evening (May 29)
* a half-day community session on Sunday morning (May 30), and
* a casual family-friendly activity in the afternoon for those who would like to stay and explore Paris together.

SDS POPS welcomes patients, families, caregivers, clinicians, and researchers to build a lasting partnership between the SDS community and the research world.

Virtual attendance will also be available for those who can't travel.

Translation support and financial assistance will be available.

We're in the early planning stages and would love to know if you're interested. Please fill out our 3-minute interest survey below — your input helps us plan the right event for the SDS community.

👉 SDS POPS Interest Form - Paris 2027 https://hubs.li/Q04sl6W90

Save the date for the congress: https://hubs.li/Q04sl1520

08/06/2026

Every August, it's back-to-school season. And with it, a new set of teachers who have never heard of SDS and don't know you or your child. 📚

This month, SDS Alliance is launching two free resources to help you introduce yourself — or your child — quickly and clearly, in a way that shows the whole person, not just the diagnosis.

* A personalized school letter tool — answer a few questions, add photos, select accommodations, and out comes a ready-to-share letter or trifold brochure. Free. No account needed. Your info stays in your browser.

* A comprehensive school guide — for patients, families, and school teams. IEPs, 504 plans, neutropenia at school, cognitive differences, transition to college and work. Applicable internationally.

Both are free. Available now.

📖 Full blog post: https://hubs.li/Q04rWH6v0
👉 School letter tool: https://hubs.li/Q04rWW8J0
👉 School guide: https://hubs.li/Q04rWP2y0

What challenge has school brought for you or your family — and what has helped? Share in the comments. 👇

07/23/2026

Big news for SDS-GPS: the data you and your family have shared with our survey research program (aka registry) is now connected to an international research platform used by scientists and drug developers worldwide.

Here's what that means in plain terms: your de-identified data (nothing that identifies you personally) now flows into a shared system alongside data from other rare disease communities and other SDS data sources in the future. Researchers use this kind of combined data to understand how SDS progresses over time and to design better clinical trials, the kind that could one day lead to approved treatments.

This has always been our vision for SDS-GPS. Every survey you've filled out, every update you've shared, adds up to something bigger than any one of us could build alone.

We wrote up the full story, including how you can join and how your privacy stays protected, on our blog: https://hubs.li/Q04qrHdM0

Thank you for being part of this. If you have questions about what data sharing means for you, drop them below.

Your SDS-GPS data just gained international reach. Learn how a new integration with RDCA-DAP is helping turn patient experience into real progress toward SDS treatments, and what it means for your privacy.

07/09/2026

We just built something new for you. 🧬

Gene therapy for SDS is advancing, and we want to make sure you understand what's being developed and who it's for. We want you to be ready to share your perspectives and needs through a survey that opens in September.

Our new interactive guide walks you through the three gene therapy approaches being studied for SDS, in plain language. No scientific background needed. Animations, visuals, and explanations at your own pace.

Watch this month's Spotlight, then explore the guide — and if you're not yet on SDS-GPS, now is the time to set up your account so you're ready when the survey opens. More details in this month's blog post.

👉 More insights in our blog: https://hubs.li/Q04n_bDv0
👉 Explore the guide: https://hubs.li/Q04n_gXP0
👉 Set up your SDS-GPS account: https://hubs.li/Q04n_Nxt0

What questions do you have about gene therapy for SDS? What excites you about the prospect of gene therapy for SDS? Drop them in the comments.

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.

06/08/2026

I'm attending the World Orphan Drug Congress in Boston this week, the world's leading Rare Disease and Orphan Drug event. Ping me if you would like to meet up while you are in town! And check out our brand-new Voice of the Patient Report for SDS at https://hubs.ly/Q04ktc7F0

06/04/2026

One year ago today, the SDS community showed up. In Cincinnati. Online. From around the world. You shared what it actually feels like to live with SDS — the fear, the exhaustion, the hope, and the things no lab result can capture.
This month's SDS Spotlight marks that anniversary — and shares what came of it.
The Voice of the Patient report for Shwachman-Diamond Syndrome is now published, submitted to the FDA, and available to anyone working to understand and treat this disease. Your words. Your stories. Now a permanent, citable document that researchers, drug developers, and regulators can use.

Watch the Spotlight, then read the full blog post for more on what the report documents and how it will be used.

Do you see your experience and priorities represented? Would you like to share more?

👉 https://hubs.ly/Q04k9K6f0
📖 Full blog post: https://hubs.ly/Q04k9Mcb0

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.

05/30/2026

This month, while celebrating the RTWF grant to support gene therapy development, we introduced you to the garden — the idea that SDS Alliance tends a whole ecosystem of research, not just one favorite project, and that patients and families are the sun that keeps it growing.

Today, we want to build on that momentum — 🌱 here is how you can help water the garden to build toward clinical trials by 2030.

We just published a free 23-page community fundraising guide — Fundraise Your Way to — for patients, families, and supporters who want to do something, on their own terms, without it taking over their lives.
It covers eight ways to get involved, from becoming a Monthly Giving Ally with a $5 monthly gift, to birthday fundraisers, t-shirt campaigns, local events, and our annual Step Up to challenge. It also includes:
💦Ready-to-use social media templates
💦 A story example and fill-in template
💦 An appeal letter you can send with your holiday cards
💦A free personalized fundraising video we will create for your family

You do not have to fundraise to support this mission. But if you have been looking for a way to do more — and want real, practical help doing it — this guide was built for you.

Read more and download it free: https://hubs.li/Q04jpvdn0

There is strength in numbers. Let's use it to . 🌱

SDS Alliance's free fundraising guide helps families and supporters raise funds for Shwachman-Diamond Syndrome research — on their own terms, at any level.

05/12/2026

I'm at Annual Meeting this week! Message me to meet up. Would love to catch up in person about all things Shwachman-Diamond Syndrome to .

American Society Of Gene And Cell Therapy, 2026 Annual Meeting, Boston, May 11-15

05/07/2026

This month’s SDS Spotlight is live — and we have big news to share. 🌱

SDS Alliance has received a significant grant from the RTW Foundation, designated for prime editing gene therapy research for Shwachman-Diamond Syndrome. In this month’s video, Eszter explains what this means, how we’re deploying it, and why our approach to research funding matters for the future of SDS therapy development.

Funding doesn’t equal impact. Funding + strategy does.

Watch the video, and read the full story at the link below.
📖 https://hubs.li/Q04fF9Fy0

What does "cure" mean to you? It means something different for everyone — and for a disease like SDS, the answer shapes how we invest in research. We'd love to hear your views.