Accelerating
life-transforming
therapies

What CureSHANK Does

CureSHANK is a research advocacy organization expediting life-changing therapies for Phelan-McDermid syndrome (PMS), a highly disabling neurodevelopmental disorder caused by pathogenic changes to the SHANK3 gene.

We work toward this goal by funding targeted research and holding scientific meetings to connect diverse stakeholders.

CureSHANK’s strategic priorities are projects that help to bridge the “translational research gap” between basic science and successful clinical trials. We identify and fund projects related to drug discovery and design, pre-clinical development, and (one day) early phase clinical trials.

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A young boy in a wheelchair
A young girl in a stroller
A young boy walking along a beach path

Our Mission

CureSHANK’s mission is to accelerate the development of treatments for Phelan-McDermid syndrome through supporting research.

We are committed to helping improve speed and efficiency in the field, so that PMS treatments get from lab to loved ones more quickly. We believe that together we can build a better future for families dealing with Phelan-McDermid syndrome and other SHANK-related neurodevelopmental disorders.

Our Motivation

The founders of CureSHANK are all parents of children affected by Phelan-McDermid syndrome. We have seen such tragedies as our children’s skills stolen through developmental regression; their brains taken over by refractory epilepsy; their bodies subject to severe hypotonia, making basic life activities exhausting; their minds overtaken by psychiatric illness. And much more. A better life for our children is within reach, and our unwavering promise is to make that a reality.

Our Values

Integrity

Our commitment to integrity means we will stay true to our mission; maintain the highest levels of transparency in our investments in science and reporting of our impact; and promote scientific excellence.

Urgency

We are working without delay to help bring patients the treatments they need.

Collaboration

We believe that collaboration is critical for the speedy and efficient accomplishment of our mission.

Our Initiatives

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    Research Funding

    CureSHANK directly funds PMS research in areas identified as particularly urgent and/or deficient. For 2023-2024, we have allocated nearly $300,000 toward research grants, the majority of which is funding the Epilepsy in Phelan-McDermid Syndrome Grant and the Developmental Regression in Phelan-McDermid Syndrome Grant. We also co-sponsor research grants with other organizations, such as the Autism Science Foundation: current collaborative research funding includes studies related to neuropsychiatric regression and sleep dysfunction. CureSHANK also supports other relevant research institutions' fundraising efforts.

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    Industry Consortium

    Our most complex endeavor is the CureSHANK Biomarkers and Outcome Measures Consortium, for which we've earmarked $500,000 for 2023-2024. This consortium is composed of companies working (or interested in working) on SHANK3 therapeutics, and this collaborative effort identifies and funds projects of mutual benefit. The Consortium also sponsors public projects useful to SHANK3 research, such as the "Resources for Researchers" webinar series.

  • Research Support

    CureSHANK supports research in many other ways besides direct funding. We recruit patients to participate in clinical research studies and clinical trials. We consult with companies about the experience, priorities, and perspectives of PMS patients and their caregivers. Recently, the CureSHANK Consortium launched our "Resources for Researchers" webinar series, and we maintain lists of Research Resources and Funding Opportunities for researchers.

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    Patient-Focused Drug Development

    Patient communities who take on projects that promote the FDA's interest in Patient-Focused Drug Development (PFDD) are attractive to companies interested in developing therapies. CureSHANK has completed several PFDD projects utilizing the expertise of PMS patients and their caregivers regarding what it is like to live with PMS, what symptoms most impact quality of life, and how families would evaluate risks and potential benefits. These projects include the PMS Conceptual Model, EL-PFDD Meeting before the FDA, and the Real Family Stories video series.

Contact Us

433 N. Camden, Suite 1100
Beverly Hills, CA 90210