A MAJOR MILESTONE FROM THE SSRA MEDICAL AND SCIENTIFIC ADVISORY COMMITTEE
Pushing the Boundaries of Rare Disease Research
This month marks a major moment for the Superficial Siderosis Research Alliance (SSRA) and everyone who supports the fight against superficial siderosis (SS).
After weeks of intense collaboration and review, the SSRA Medical Advisory Committee officially submitted an FDA research grant application to accelerate progress toward a safer, more effective treatment and, ultimately, improved quality of life for SS patients.
This submission isn’t just a step forward. It’s a leap; the result of dedication, teamwork, and the shared belief that rare disease research deserves the same urgency and attention as any other condition.
Inspired by the 2025 Research Symposium Roundtable
Following the success of our 2025 SSRA Research Symposium Roundtable (read about it here), our medical advisory committee found new momentum. What began as quarterly meetings quickly transformed into weekly working sessions for six straight weeks, all focused on identifying the perfect research opportunity to build upon the ideas and collaborations sparked during the symposium.
That opportunity arrived in the form of an FDA research grant that aligned perfectly with our mission and research needs.
The Grant Proposal: Two Studies, One Goal
After serious discussion on what our patient community had shared were their primary goals, it was agreed that our grant proposal would combine two previously developed research concepts into a single, streamlined plan:
- An open-label clinical trial to evaluate the safety profile of deferiprone, an iron chelator currently used off-label by some SS patients.
- A natural history study for patients who prefer to contribute data without taking deferiprone.
Together, these complementary studies will help researchers answer two vital questions:
- Is deferiprone safe for long-term use in superficial siderosis?
- Can it meaningfully slow disease progression and improve quality of life?
If awarded, the grant will allow eligible patients in the United States and the United Kingdom to participate in either study. Participants in the clinical trial arm will receive deferiprone at no cost for up to four years. Meanwhile, data from the natural history study will help researchers better understand the disease’s natural progression, information that could prove invaluable for future treatment development.
Why an FDA IND Application is the Next Step
Before any clinical trial can begin, the research team must secure FDA approval through an Investigational New Drug (IND) application.
The IND ensures that:
- The proposed study is scientifically sound.
- Patient safety is prioritized through well-designed monitoring and reporting systems.
- All research materials, from the drug formulation to consent forms, meet regulatory standards.
SSRA Research Committee member Rori Daniel is now finalizing and polishing the study protocol for IND submission, aiming to ensure the trial meets the FDA’s high expectations for scientific rigor and patient protection.
What This Means for the SS Community
For patients, caregivers, and advocates, this grant submission represents something powerful: momentum.
Research on superficial siderosis has long been limited by funding and the rarity of the condition. But this proposal and the collaborative energy behind it reflect a turning point.
If the grant is awarded, it would not only fund the combined trial but also build the foundation for future studies aimed at moving deferiprone from an off-label treatment to an FDA-approved therapy. That change could make access easier, safer, and more affordable for patients around the world.
Looking Ahead: Next Steps and Timeline
Dr. Michael Levy submitted the grant application and 178-page study through Massachusetts General Hospital before the official deadline, a race that required incredible focus and teamwork by Advisory Committee members, Rori Daniel, and Dr. Natallia Kharytaniuk. The SSRA expects to receive initial updates from the FDA and funding committees in April or May.
In the meantime, the organization continues to refine the research plan, secure additional support, and prepare for potential next steps once approval is granted.
How the SSRA Continues to Support the Mission
The SSRA remains committed to funding any research needs not covered by the grant. Every donation, every volunteer effort, and every show of support helps bridge critical gaps that federal grants can’t always fill.
A Shared Vision for the Future
While this grant application is a technical milestone, it’s also deeply personal. Every page of the proposal represents the stories, challenges, and hopes of the SS community, the patients living with progressive neurological symptoms, the caregivers providing constant support, and the doctors working tirelessly to find answers.
The SSRA’s vision is clear: a future where superficial siderosis can be treated safely, effectively, and compassionately.
And this grant brings us one step closer to making that vision a reality. The SSRA thanks all medical advisors and supporters who made this submission possible. Together, we’re driving research that matters.
Stay Connected
We’ll share more updates as the review process moves forward in the coming months.
Want to support ongoing SS research? Consider contributing to the Superficial Siderosis Research Alliance or joining our advocacy efforts during Rare Disease Week 2026 (learn more here).
Together, we’re proving that even the rarest conditions deserve powerful, persistent research.
External Links:
- Inside the 2025 Research Roundtable – SSRA Symposium Highlights
- EveryLife Foundation – Rare Disease Week 2026 Agenda
