For Academia
& Industry
CureSHANK is helping bridge the "translational research gap" between basic SHANK3 science and successful clinical trials. We identify, support, and fund strategic projects useful to industry and academic investigators alike who are interested in—or already pursuing—this goal.
Patient-focused Drug development
Patient-Focused Drug Development (PFDD) is a crucial ingredient in the process of developing and evaluating therapeutics. As Food and Drug Administration (FDA) explains, the PFDD approach helps ensure that patients’ experiences, perspectives, needs, and priorities are captured and meaningfully incorporated into drug development and evaluation.
To that end, CureSHANK has organized and funded several projects that both keep Phelan-McDermid patients’ high level of unmet need front and center, and also fulfill FDA’s requirement that such a focus be incorporated in the development of treatments.
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PMS Conceptual Model
In 2022, CureSHANK completed the Phelan-McDermid Syndrome Conceptual Model. This project surveyed fifteen peer-reviewed publications, categorizing nearly 100 signs and symptoms of PMS, as well as observer-reportable patient and caregiver impacts. CureSHANK invested $20,000 to produce the Conceptual Model.
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EL-PFDD Meeting
Nearly 300 families participated in the Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting for Phelan-McDermid Syndrome in November 2022. It was a groundbreaking opportunity to share the patient and caregiver experience of PMS, and it provided crucial context for the FDA to consider when evaluating requests for clinical trial approval.
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Real Family Stories
CureSHANK’s Real Family Stories video series highlights the lived experiences of families caring for individuals living with Phelan-McDermid syndrome. These videos are available for use by anyone seeking to demonstrate the high level of unmet need in the PMS population.
Cureshank Consortium
The CureSHANK Biomarkers and Outcome Measures Consortium facilitates a cooperative, globally aligned effort focused on the identification and development of effective approaches to enable successful clinical trials for Phelan-McDermid syndrome. CureSHANK has committed $500,000 to the Consortium thus far.
Resources for Researchers
CureSHANK connects researchers with funding and resources not only through CureSHANK-sponsored projects, but also by maintaining a thoughtfully curated list of funding opportunities and research resources available to researchers engaged in both basic SHANK3 science and clinical research.
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Funding Opportunities
In addition to information about CureSHANK Research Grants, we maintain a curated list of funding opportunities of interest to SHANK3 researchers, because we know very well that research cannot happen without funding. We also reach out to our interested researcher list from time to time with more specific grant opportunities.
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Research Resources
One key way CureSHANK’s is helping bridge the translational research gap between basic SHANK3 science and treatments for Phelan-McDermid syndrome is by connecting researchers with resources. We have gathered a list of useful resources and have also launched a “Resources for Researchers webinar series.