For Patients

Partnerships for Rett Syndrome

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Rett Syndrome Research Trust

The Rett Syndrome Research Trust is a nonprofit organization solely focused on development of a cure for Rett syndrome. Their current focus is on the implementation of six genetic-based strategies and the funding of research worldwide to achieve this goal.

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International Rett Syndrome Foundation

The International Rett Syndrome Foundation is a leader in the acceleration of research to treat and cure Rett syndrome. Having supported almost 40 years of progress, their foundation has impacted the Rett Syndrome population tremendously through funding that has led to, but not limited to, the discovery of the gene that causes Rett syndrome, the first gene therapy research in Rett syndrome, and the development of the first clinical trial for Rett syndrome, which was conducted at Boston Children’s Hospital.

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Rett Syndrome Angels

Formerly the Rett Syndrome Association of Massachusetts (RSAM), this New England-based organization provides support to Rett syndrome families by providing services, resources, awareness, and fundraising for the advancement of Rett syndrome research and improving the lives of those impacted by Rett syndrome.

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CLINICAL TRIAL

An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome (DAFFODIL™)

PI: David Lieberman, MD, PhD  |  Clinical Drug Trial — NCT04988867 >
Patient Advocacy Group Partner: International Rett Syndrome Foundation (IRSF)

CLINICAL TRIAL

Assessing Emerald and MC10 Biostamp nPOINT Biosensor for Rett Syndrome

PI: David Lieberman, MD, PhD  |  Observational Device Trial — NCT04514549 >
Patient Advocacy Group Partner: Rett Syndrome Research Trust