In this issue: SDSF and SDS Alliance are excited and honored to announce that an Externally-Led Patient Focused Drug Development Meeting for Shwachman-Diamond Syndrome has been accepted by the FDA and is planned for June 4th, 2025 in a hybrid format (Zoom and in-person in Cincinnati, OH). Apply to be a Patient or Caregiver Speaker/Panelist today!
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!
Dear patient/caregiver,
SDSF and SDS Alliance are excited and honored to announce that an Externally-Led Patient Focused Drug Development Meeting for Shwachman-Diamond Syndrome has been accepted by the FDA and is planned for June 4th, 2025 in a hybrid format (Zoom and in-person in Cincinnati, OH).
EL-PFDD meetings give the FDA and other key stakeholders, including medical product developers, healthcare providers, and federal partners, an important opportunity to hear directly from people living with SDS, their families, caregivers, and patient advocates about the symptoms that matter most to them, the impact the disease has on people’s daily lives, and their experiences with currently available treatments. This input can help inform the FDA’s decisions and oversight during drug development and new drug review, which is crucial for expanding options for improving the lives of those living with SDS. It will also help medical product developers, researchers, clinicians, and the general public gain an in-depth understanding of what living with SDS really means.
The EL-PFDD meeting is a once-in-a-lifetime opportunity for you—patients and caregivers—to use your voice and make an impact on the future of SDS therapy development. It is an opportunity to share your experience and perspectives on living with SDS, particularly as they relate to the community's unmet needs and consideration for future therapy development efforts. Learn more on the EL-PFDD for SDS information page.
There will be several ways and opportunities for you to contribute your voice, from speaking at the EL-PFDD meeting live or virtually, responding to polls and discussion during the meeting, filling out surveys ahead of the meeting, and participating in a qualitative research interview (SDS Patient LENS study).
Right now, we are specifically looking for patient and caregiver speakers/panelists for the EL-PFDD meeting. Applications are now open through January 26, 2025. Apply using this survey. Selected speakers/panelists will receive support in developing and delivering their stories through professional coaches, and their travel/accommodation will be covered. Childcare may be available upon request.
Please note that the EL-PFDD meeting is scheduled to take place on the day before the SDS Congress (11th International Schwachman Diamond Syndrome Scientific Congress in Cincinnati, OH), at the same or nearby location (and via Zoom). While we are coordinating logistics with the SDS Congress organizers, the two events are independent of each other. Registrations don’t carry over from one to the other.
We look forward to hearing from you about how you would like to contribute to the EL-PFDD meeting to elevate the patient voice and use it to drive change and improve therapeutic options.
Sincerely,
Eszter Hars
Mother of a child with SDS
Founder and CEO, Shwachman-Diamond Syndrome Alliance
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