Thank you for watching SDS-POPS 2025, the global Shwachman-Diamond Syndrome Patient AdvOcacy and Partnering Summit. As we continue to expand the SDS Alliance’s programming and engagement opportunities, your feedback is very important in shaping our work moving forward. Please fill out this anonymous 5-10 minute survey to share your experience with the SDS-POPS team. We will only ask for your name and contact information only if you wish to join our mailing list or request a follow up.
How would you rate the following aspects of the summit on a scale of 1 to 5 (5 being the highest positive score)?
The overall experience with the summit.
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Session I: Therapeutic Modalities: Focus on Gene Therapy? Have the topics met your expectation?
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Session II: How can we - as patients and families - make a difference? An overview of how to move a therapy from the lab to actual patients. Have the topics met your expectation?
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Scientific talks by guest (during session I): Was the content level right for you?
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Framing/overview of the scientific talks (by Eszter): Was it helpful for your understanding of the scientific talks?
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level of helpfulness
SDS POPS meeting technical aspects. Was it easy for you to access and navigate the meeting?
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Would you like to share more thoughts on any of the above?
Did you join SDS-POPS real-time through Zoom, or watched the recording at a later time?
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Joined SDS-POPS real-time through Zoom
I didn't have time for either options, but plan to watch the recording in the future
Watched the recording later
Attended both real-time AND watched the recording (or parts of it)
Other
Have you used any of the translation tools available to access the meeting to content in a language other than English?
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I only accessed the contents in English
I used Zoom's caption translation tool (while attending in rea-time)
I used YouTube's automatic caption translations (while watching the recording)
I asked a friend/family member to translate for me
Other
Which language did you use? How did the translation method work for you?
How did you hear about the POPS Summit? Select all that apply.
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Facebook
X (Twitter)
LinkedIn
WhatsApp
SDS Alliance email/Newsletter
From a friend/family
From a colleague/speaker
From my doctor/HCP
Other
Based on SDS POPS, or what you already knew about the SDS Alliance's mission, what is your understanding of that the SDS Alliance does for you?
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Analyzes the whole landscape of SDS therapies to determine what and how to support to take therapies all the way to patients.
Invest time, effort, and funding into research tools to create critical tools and make them accessible to any and all researchers who may need them
Invest time, effort, and funding into research infrastructure, such as bringing the patient voice to the FDA and other regulators. This paves the path to regulatory approvals of SDS therapies in the future.
Got a US ICD-10 code for SDS to create real world evidence and make the patient journeys in EHR visible.
Runs a survey platform - SDS-GPS - for SDS patients and families to share their lived experiences and learn about relevant clinical trials. Available in 5 languages, secure, IRB approved, GDPR compliant.
Created a biobank for patient-derived cells for researchers to access easily from anywhere.
Actively works with collaborators to develop mouse models for SDS, persisting through technical challenges where previous efforts have given up
Stuards donations by the SDS community for maximum impact toward accelerating therapy development. Instead of fundraising for a specific researcher or institution, SDS Alliance actively develops collaborations and funds projects based on timelines and deliverables to ensure maximum impact. This paves the path to more collaborations in the future, and offers flexibility to support what is most needed as the needs and landscape evolve.
Our strategic goal is to make multiple Clinical Trials for SDS by 2030 a reality. We focus on projects that are the closest to the clinic, as they are most likely to benefit patients the soonest, and will open doors to other therapies as well. We need all shots on goal, as one therapy will not meet all patient's needs.
Makes family fundraising easy, fun, and meaningful, in order to maximize the impact of the community's efforts
Creates opportunities for patients and families to engage with researchers for mutual learning and collaboration.
Created a documentary film to highlight SDS to a broad audience and move the hearts and minds of researchers and regulators.
Other
What is your connection to Shwachman-Diamond Syndrome (SDS)?
Please select all that apply.
I am
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an adult diagnosed with SDS
a parent/primary caregiver to someone diagnosed with SDS
suspecting or pursuing an SDS diagnosis for myself or a loved one
a friend/family member of someone with SDS
a researcher interested in SDS
a healthcare provider interested in SDS
a diagnostic provider for SDS
an advisor/mentor to the SDS Alliance
sponsor/grant maker to the SDS Alliance
a service provider/vendor to the SDS Alliance
a nonprofit representative from a related/rare disease community
other
If other, please explain
Your Primary Language. (We aim to offer our resources in multiple languages in the future.)
Please Select
English
Spanish
German
French
Italian
other
If you selected 'other', please enter your primary language here.
Adult Patient
Adult Patient
Caregiver
Caregiver
Friend/Family
Friend/Family
Pursuing Diagnosis
Pursuing Diagnosis
Research Network Member
Research Network Member
Clinician Network Member
Clinician Network Member
Nonprofit Rare Disease Community
Nonprofit Rare Disease Community
Industry Advisor
Industry Advisor
Mentors, Partners, Peers
Mentors, Partners, Peers
Volunteer (current or former)
Volunteer
Sponsor/Grant Maker/Business Partner
Sponsor/Grant maker/Business Partner
Service Providers/Vendors
Service Providers/Vendors
Diagnostic Providers
Diagnostic Providers
Do you have comments/suggestions on how we can improve future SDS-POPS and other meetings? This can include the logistics, contents, timing, or any other aspect that matters to you.
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Please share some general feedback (testimonials) we can use to share with the community to encourage families and professionals to join future events. Or share any other feedback you would like to share with the SDS-POPS team.
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We are developing and refining a variety of information resources for both the patient community and the medical community. What topics would be most helpful to you and your care team?
Genetic testing (why it's needed and how to access it)
Bone Marrow Surveillance (to asses whether a transplant is warranted)
Pancreatic Exocrine Insufficiency, Feeding, and Nutrition (including pancreatic enzyme replacement therapy PERT)
Vaccinations (general considerations and recommendation for SDS patients)
Neurocognitive and developmental assessments (recommendations for SDS patients)
Liver issues in SDS
Dental and oral challenges (tips and recommendations)
Infections and fever protocol
Registries and biobanks as a resource for patients and researchers
Support resources for patients and their families
Stem cell transplant in SDS (general guidance and factors to consider)
Growth and Skeletal issues in SDS
Diabetes and glucose regulation in SDS
Growth charts and challenges in SDS
School resources and special education support
genetic testing resources
Fertility and reproductive health
Other
What services would you or your family like to access? Please select all that apply.
Connecting (1:1) with other SDS patients and families who have similar experiences as you, live near you, and/or speak the same language
Receiving a welcome package designed for families who are new to the SDS Alliance community (including simplified information about SDS and the SDS Alliance, support resources, and comfort items lovingly hand-selected by our volunteers)
Finding healthcare providers with SDS expertise in my area
Watching webinar recordings or short videos featuring SDS experts and peers
Accessing written educational resources regarding SDS science, research updates, and the therapy development process
Participating in interactive webinars featuring SDS experts and peers
Accessing educational resources regarding clinical care, diagnostics, and support services
Receiving relevant updates about clinical trials and other research opportunities
Joining virtual peer support and community groups for SDS families through video calls
Joining virtual peer support and community groups for SDS families on Facebook or other platforms
The SDS documentary film "Until There's a Cure"
Fundraising tools and help
Other
Do you have requests or ideas for new programming the SDS Alliance team should explore and maybe offer in the future?
Would you like to learn more about specific projects/research opportunities highlighted at SDS-POPS? Or join our mailing list?
We will only ask for your contact information if you do.
Yes, please tell me more about the following opportunities
SDS-GPS: Global Patient Survey and Collaboration Program by the SDS Alliance. Now open to all patients and caregivers, and available in five languages!
4WARD Study by X4 Pharma
Registries and Natural History Studies available in my area
Genetic testing opportunities for my family or relatives
Community connection and support opportunities
Upcoming fundraisers (i.e. Million Steps Closer to #CureSDS)
Global SDS Action Week activities and opportunities
Project PACER, to learn more about SDS and effective partnerships in all phases of research
The SDS documentary film "Until There's a Cure"
SDS POPS recordings
Other
Would you like to join the SDS Alliance and Global Network mailing list? You can unsubscribe or update your preferences anytime using the links in the footer of emails I receive from the SDS Alliance.
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Yes, please add me to your email list and Global Network.
No, don't add me to your email list. (This won't remove you if you joined in the past).
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