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About RSRT

At RSRT we are relentless in our dedication to advancing the research to a cure. This is our goal. This is our passion and our obsession. We won’t rest until we achieve it.

For us it’s personal. Our chief executive officer, chief development officer, and family liaison all have a child with Rett. Twelve of our board members have a child, grandchild, or sibling with Rett syndrome. We are driven by the most profound motivator — love for our children and an unwavering commitment to change their lives and the lives of all with Rett. Backing this up are decades of experience in each of our fields.

Our scientific staff are equally dedicated. They are driven by a passion for science and a zeal to translate that science into life-changing cures. Their knowledge, their integrity, and their combined experience of over 60 years in drug development and clinical trials fuel their commitment to reaching our goal.

RSRT’s unique fusion of dedication, passion, knowledge, and experience gets results:

  • $62 million awarded to research since RSRT’s founding in 2008 — more than any other Rett organization anywhere.
  • Incubated curative academic research programs successfully transitioned to biopharma. Every single company pursuing a cure for Rett is able to do so because they leveraged discoveries and resources that were incubated with RSRT funding.
  • An organization that funds the boldest research that other organizations might shy away from. We don’t aim for palliative; we aim for a cure.
  • A reputation as an organization that’s direct and honest about research results. Spin and exaggeration are unacceptable.
  • An unparalleled commitment to efficiency and integrity; an average of 95% of every dollar donated to RSRT goes directly to our research program.
  • An operation and structure that leads rather than reacts. RSRT does far more than just raise funds and then write checks. RSRT engages every day with researchers, biopharmaceutical company leaders, and clinicians and works with them to set the research agenda.
  • A powerful sense of unity and aspiration in the Rett community; together we CAN do this. Thousands of families take action and fundraise to make our research possible.

We do not rest for one minute on our achievements thus far. Our ultimate goal is on the horizon. We invite you to join us as we push onward to reach that goal.

CURE 360

From 2017 to 2020 we carried out Roadmap to a Cure, a research plan that identified and advanced six therapeutic strategies targeting the root cause of Rett syndrome.

CURE 360 is the blueprint for how we now drive those advances into a coordinated, full-on attack on Rett.

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Rett Timeline

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    1966
    1st article published on Rett in German medical journal
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    1983

    2nd article on Rett published in high-profile neurology journal

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    1992
    MECP2 protein discovered by Adrian Bird
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    1999
    MECP2 mutations identified by Huda Zogbhi as cause of Rett
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    2007
    Adrian Bird discovers Rett symptoms in mice are reversible
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    2008
    Rett Syndrome Research Trust launches
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    2011
    MECP2 Consortium launches
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    2013
    Gail Mandel and Brian Kaspar publish gene therapy results
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    2014
    RSRT Gene Therapy Consortium launches
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    2017
    Roadmap to a Cure launches
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    2017
    Clinical Trial Consortium launches
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    2018
    Jim Wilson joins gene therapy effort
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    2019
    Beam joins DNA editing effort
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    2020
    CURE 360 launches
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    2020
    Novartis confirms commitment to gene therapy program
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    2020
    Taysha announces gene therapy program
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    2020
    Vico Therapeutics announces RNA editing program
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    2020
    Shape Therapeutics announces RNA editing program
  • Alcyone Therapeutics
    2021
    Alcyone Therapeutics announces lead MECP2 reactivation program
  • Herophilus
    2021
    Herophilus announces lead MECP2 reactivation program

Rett News

Visit RettNews to view our press releases along with the latest news on Rett syndrome.