A benchmark goal of the Marshall-Smith Syndrome Organization of the USA is to create a patient registry for MSS. We plan to create this registry through the IAMRARE program offered through the National Organization of Rare Diseases (NORD).
The IAMRARE program was created with input from the FDA, NIH, patients, organizations, and experts in the field. Having a natural history of MSS and registry of patients around the world will give more opportunities for critical research.
As a part of the IAMRARE program, our organization will have more control and oversight of the research being done on Marshall-Smith Syndrome. By consolidating patient-generated data in a central location we can increase what is known about MSS, improve standards of care, and have a "seat at the table" for any clinical or pharmaceutical research opportunities.
MSS-USA will need to raise $15,000 to create our natural history patient registry through the IAMRARE program. Our goal is to have this fully-funded and begin the process by June 2024.
"NORD’s IAMRARE Program is an easy to use system that allows patients and organizations to inform and shape medical research and transnational science for rare diseases by launching high-quality, customized registries to collect the data needed to define the natural progression of their disease – ultimately advancing product development."
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