List of all posts in this site

Virtual EVAning of Hope 2021

Happy Hour to Help End Rett

Novartis OAV201 Update

Cocktails for a Cure: A fun, social way to raise funds for the research during the pandemic and beyond!

Virtual Reverse Rett Chicago 2021

Miracle for Molly

Reverse Rett Austin 2021

Virtual Reverse Rett Philadelphia 2021

Reverse Rett NYC 2021

Imagine Revolutionizing Your Child’s Clinical Care – Introducing the Rett Syndrome Global Registry

Reverse Rett LA 2020

Reverse Rett NYC 2020

Speechless. For Rett.

Form Test

The Next Phase of Our Attack on Rett: CURE 360

AveXis/Novartis Statement on AVXS-201

It Will Consume Your Life: 4 Families Take on Rare Diseases

Enhancing the Potential Impact of Gene Therapy

Fundraising in the Time of COVID-19

Mutations Fighting Mutations

The Power of Three

Rett Research in a Time of Crisis

Biomarkers – What are they and why do we need them?

The Launch of a New Partnership

Ketamine Clinical Trial

1. Why should I consider participating in the ketamine study?

2. Who is eligible for the ketamine study?

3. What will happen during the study and what do I and my child have to do?

Can we test length?

How will a second question work?

Does this work?

2019 Research Awards | RSRT

Can Food be Medicine?

Exciting News for the MECP2 Duplication Syndrome Community

New Insight Into How MeCP2 Regulates Transcription

Reverse Rett Washington DC 2020

The Gift of Giving

Prime Time

Pride (In the Name Of Love)

Awareness is Not Enough

Reverse Rett Mountaineer Tailgate

Reverse Rett Boise 2020

Virtual Quest for a Cure Walkathon

AveXis/Novartis Release Statement on AVXS-201

Biosensors and the Future of Rett Therapeutics

Harnessing Exosomes as a Therapeutic for Rett Syndrome

New Drugs Restore Brain Balance to Treat Rett Syndrome

Let’s Flamingle for a Cure

If Not Us, Who?

My First 9 Months at RSRT

2018 Annual Report | RSRT

Reverse Rett Philadelphia

Reverse Rett Upstate New York

Making Sense of it All

A Big Step for Gene Therapy

Reverse Rett Minneapolis

Miracle for Molly

Reverse Rett Chicago 2020

Reverse Rett Cape Cod 2020

Cure MECP2 Bucks County

Ketamine Clinical Trial for Rett Syndrome Launches

Gene Therapy Consortium 1.0

Reverse Rett Austin 2020

Reverse Rett San Antonio 2020

RSRT $10 Million to Preeminent Researchers in Pursuit of Curing Devastating Neurological Disorder

On the Shoulders of Giants

2018 Research Awards | RSRT

Three Stars and
Prouder than Ever

What’s Making MeCP2 Toxic in Duplication Syndrome?

Rett Superheroes

World-Class Team Takes on Next Gen Gene Therapy

Take Action

New Method Developed to Study MeCP2 Function in Humans

Join A Committee

Paul Temp

Are You In?

Talk To Your Doctors

Reverse Rett NYC 2019

What Does it Take to Run a Clinical Trial?

UPDATE: AVXS-201 Gene Therapy for Rett Status

Moving the Needle

Confessions of a Novice Event Planner

For Love of Lily

Reverse Rett Brisbane

Reverse Rett LA 2019

California Raisin’ for Rett

Happy Hour to Help End Rett 2019

Reverse Rett DC 2019

RSRT Names Timothy A. Riley Chief Scientific Officer and Chief Business Officer; Randall Carpenter Transitions to Chief Medical Officer

Quest for a Cure 2019

Mack’s Crabs & Cornhole for a Cure

Reverse Rett Boise 2019

Reverse Rett Dallas

The Future is Now

EVAning of Hope

Let’s Flamingle for a Cure 2019

We’re in This Together

Meeting of the Minds

Reverse Rett Chicago 2019

Miracle for Molly 2019

Unapologetically Going for the Cure

Preparing for a Clinical Trial

Igniting Hope

Scientists are thinking the unthinkable: CRISPR might one day reverse devastating brain diseases

Novartis to Acquire AveXis

Reverse Rett Minneapolis 2019

French Translations

Reverse Rett Kansas City

Cure MECP2 Bucks County 2019

Drug Development Is Cheap And Easy, Said No One Ever

Making It Count

Home Team Advantage

A Rigorous Peer Review: Why You Should Care

Vision and Guts

German Translations

Reverse Rett Austin 2019

AveXis Reports on Rett Gene Therapy Program: AVXS-201

Funding Collaboration Not Competition

Spanish Translations

Reverse Rett San Antonio 2019

RettGive Maintenance

The Road to FDA Approval

Rides for Rett

Reverse Rett Litchfield

Is a Cure for Rett Really Around the Corner?

2017 Research Awards | RSRT

Pioneering Scientist Joins Gene Therapy Consortium

The Man Behind the Brand

Inspired by You

Cocktails for a Cure

$14,230,348 and 2 cents

Preparing for Gene Therapy Trials

Breakthrough Discoveries for DNA & RNA Editing

Reverse Rett NYC 2018

Editing Out Rett Mutations

Reverse Rett Utah 2018

RettGive: Easy, Impactful, Empowering

Jocelyn’s Journey

Happy Hour to Help End Rett 2018

Reverse Rett LA 2018

Bridging the Divide

October: Rett Awareness and So Much More

Social Graphic Creator

How Rett Changed a Granddad

Quest for a Cure 2018

Reverse Rett DC 2018

Roadmap Update: Jaenisch Lab Award

Twice the Heartache…Twice the Love

Reverse Rett Melbourne 2018

Marlowe’s Web

The Rollercoaster of Rett

Rohan’s Quest 2018


Thanks For second Donating

Thankyou For Donating

Sample Page

2016: A Year in Review

Reverse Rett Philadelphia 2018

What About Us?

Finding the Words

Precision Medicine

AveXis Announcement – Delivering on Roadmap to a Cure

Reverse Rett Chicago 2018

Gene Therapy Consortium Reports on Progress

Gene Therapy Primer

Out Run Rett 5K

Miracle for Molly 2018

Racing Down The Gene Therapy Highway To A Cure

Reverse Rett Minneapolis 2018

Orphan Drug Designation: What Does it Mean?

Reverse Rett Kansas City 2018

New Insight Into MECP2 Mutation Hotspot

Roadmap to a Cure FAQ


RSRT Awards $7.5 Million to Research in 2016

Key Questions to Ask When Considering Clinical Trials

Reverse Rett Austin 2018

Awaken and Activate the Mighty X Chromosome

Of Mice and Rett: Novel Therapeutics & Community Support

Cocktails for a Cure 2018

Small Town, Big Heart

Art for Ruby 2017

The Joy and Ache of the Holidays

Rare Disease Drug Approval Sparks Controversy

The Phone Call

Gene Editing Shows Potential for Treating Genetic Diseases Like Rett

RSRT Rett Fact Cards

Rett Fact Cards

Rett Fact Cards

RettGive Spotlight: A Father’s Day & Birthday

Reverse Rett NYC 2017

Chinese Translations

Event Spotlight: Happy Hour to Help End Rett

Jocelyn’s Journey 2017

Reverse Rett LA 2017

Rett Syndrome Awareness Month 2016

Happy Hour to Help End Rett 2017

What the Chan Zuckerberg Initiative Means for Rett Research

A Quick Look Back and a Major Look Forward

For Love of Lily 2016

Cure MECP2 Bucks County 2017

Rides for Rett 2017

Mortgaging the House

Deep Brain Stimulation: Next Steps

Deep Brain Stimulation and MECP2

Ask Gaby: Episode 1

Results of Copaxone Open Label Trial

Rett Give: Are You In?

The Boys of Rett

Alba Tull Joins RSRT Board of Trustees – Press Release

Ask Dr. Carpenter: Episode 1

Ask Dr. Carpenter

EVAning of Hope 2017

Register Now

Funding in the Pursuit of Balance

First Impressions

New Trustee Appointed

If You’re Going Through Hell, Keep Going

Crowdfunding for Rett

Three Stars and Proud

The Day My World Stood Still

Reverse Rett Chicago 2017

Attacking Rett Where it Lives | More Funding Awarded

Out Run Rett 5K 2017

Mother’s Day…A Time to Bear Witness

You Don’t Know Bo?

Professor Bird Elected to National Academy of Sciences

Gene Therapy Might be the Best, and Perhaps Only, Chance at Curing Brain Diseases

Clarifying the “Vanderbilt Breakthrough Story”

Miracle for Molly 2017

“Disease in a Dish” Platform Receives a $1 Million Influx from RSRT

Everything for Emma

Modern Family

Educating the FDA on Breathing Problems

Human Gene Therapy Commentary | Monica Coenraads

YouTube Donation Cards: Every Bit Counts



Rett Syndrome Clinic in Atlanta Launches Clinical Trial

8th Grader Inspires with Rett Fundraising

$9 Million Awarded to Research

Notes from a Doting Aunt

A Word From Randy

Rett Hand Splints: What’s the Verdict?

Meet Our New CSO – Randy Carpenter

Book Review: Orphan – The Quest to Save Children with Rare Genetic Disorders

Rett in Review: Crossing Thresholds

KCC2 and Rett: The Import/Export Business

Dear Rett: Be Afraid – Be Very Afraid

It’s All About the Sneakers

The Growing Toolbox of Rett Mouse Models

Rett Researchers Gather in Boston

MECP2 Consortium Progress Report

Support RSRT While You Shop

Ketamine Trial is Recruiting

Gene Therapy Consortium Progress Update

Cheers for Charlotte

RSRT is Giving Away a Tobii C12!

MECP2 Duplication Syndrome is Reversible

Breaking a $6 Million Fundraising Record this Year!

Einstein Magazine Feature on Rett Syndrome

Reverse Rett NYC 2016

Jammin’ for Jaycee

Reverse Rett LA 2016

More About Rett

Thank You for Your Donation!


Jonathan Epstein

Reverse Rett Today

Jason M. Rothschild


Deep Brain Stimulation – A Potential Therapeutic for Rett Syndrome?

David I. Scheer

Dmitri Smolansky

AJ Tesler

James S. Trainor, Jr.

News Page Header – Don’t Delete

Events Page Header – Don’t Delete

Urological Issues in Rett

Happy Hour to Help End Rett 2016

List all posts

Financials, Press Releases, and Policies

International Clinics

Gene Therapy Consortium


Dive Deeper

Clinical Trials

About MECP2


Take Action

Newly Diagnosed

About Rett

About Us


Jocelyn’s Journey 2016

Quest for a Cure 2017

Seven Years and Counting

Latest MECP2 News from the Mandel Lab

Cure MECP2 Bucks County 2016

RSRT Awards $337,336 to Michela Fagiolini at Boston Children’s Hospital

Rett and Pharma… What’s the Attraction?

Rides for Rett 2016

Adrian Bird | Hope for Rett Syndrome

Sir Adrian Bird, PhD

Stephanie Bohn

Monica Coenraads

Tim Freeman

J. Michael Bishop, Ph.D.

Timothy Riley, PhD

Michael E. Greenberg, Ph.D.

Randall Carpenter, MD

Heidi Epstein

Franz F. Hefti, Ph.D.

Ingrid Harding

Jana von Hehn, PhD

Sharon Lynch

Nathaniel Heintz, Ph.D.

Lawrence Mattis

Jennifer Endres

Rachel Rothschild

Ricki Davis

David M. Katz, Ph.D.

Christopher Lipinski, Ph.D.

Anthony P. Schoener

Gail Mandel, Ph.D.

Rachael Stevenson

Marc Tesler

Alba Tull

Marci Valner

Brian Whitmer

Brad Zelinger

Huda Zoghbi, M.D.

LI Scientists Find Possible Treatment for Rett Syndrome

RSRT 2014 Annual Report

Super-Genes to the Rescue?

RSRT Awards $400,000 to Mark Zylka for Long Genes Screening

New Technology Crispr-Cas9 Fuels Genome Editing Conversation

Gut Microbiomes & Rett: What’s the Connection?

Gene Therapy Consortium Meeting – A Parent’s Perspective

RSRT Awards $530,000 to Neurolixis for Clinical Development of NLX-101

Rohan’s Quest for a Normal Life 2016

Studio 54 | Black & White Party

Chicago Hope

Reverse Rett Chicago 2016

Monica Coenraads Awarded Honorary Doctoral Degree – UMass Medical School

New Study Yields Conflicting Results

Bella’s Ball

Out Run Rett 5K 2016

Second Chance Masquerade

Art for Ruby 2016

Miracle for Molly 2016

Concert for Rett

Research from Harvard Points to Potential New Avenue for Treatment

Interview with Former Director of the FDA Office of Orphan Product Development, Timothy Coté

GP2C Disney Princess Marathon

California Family Sets Record for Online Giving Campaign for RSRT

RSRT Awards $5.8 Million For Research and Two Clinical Trials

We Need 9 More!

MECP2 Consortium Update – Breaking Down the Walls

Windfall: Good or Bad?

Rett and the Gut

MECP2 Gene Therapy Consortium Update

Lessons from the Ice Bucket Challenge

7th Annual Reverse Rett NYC

Sadie | Make Rett History

Reverse Rett LA 2015

A Promise

The X Factor – Michael Green Interview

Guest Blogger: Drug Therapies and Rett

Rett Syndrome: Why Girls?

It’s Up To Us

Wake Up MECP2!

Transparency in Clinical Trials

UMASS Highlights Rett and the Work of RSRT

Chasing Cures: Reinventing the Business of Medicine

Peer Review and Impact Factors

Diagnosis: Rett Syndrome

On the Hunt for Modifiers

Shaking World Views

RSRT Launches the MECP2 Gene Therapy Consortium with a $1.5 Million Investment

What’s the Buzz About CRISPRs?

RSRT Trustee, Professor Adrian Bird, Knighted

Two RSRT-funded Projects Included in Top Ten Most Notable Papers of 2013

One Person Many Genomes

Cholesterol and neurological disorders

RSRT-funded Study Shows Gene Therapy Can Reverse Rett Symptoms in Mice

2013 Scientific Investments To Date

RSRT-funded Study Shows Cholesterol-Lowering Drugs Suppress Rett Symptoms

Problems Plague PTC Therapeutics Drug, Ataluren

Welcome Tim!

The Rett Syndrome Protein Surrenders Some of its Secrets

Research Nuggets

Fragile X News


VIDEOS: “Curing Rett Syndrome – How Do We Get There?”

Learning from Patients

Boys With Rett Give Us Clues About MeCP2’s Function

Rett Researchers Get Up Close and Personal

Funding Research – Funding Results: Record $4.2 Million Awarded

A New Mode of Action for MeCP2

Who’s In Charge?

NIH Funding Woes

Four Challenges in Rett Translational Research

Innovation Meets Experience

Rett Mice Continue to Reveal Their Secrets

Podcast Interview with David Katz, PhD: Rett Syndrome and Ketamine

Life Cycles and MeCP2

Treating Synaptic Dysfunction

Bone Marrow Transplants – Proceeding with Caution

Bone Marrow Transplant Stops the Development of Symptoms in Model of Rett Syndrome

BioWorld Insight feature article on Rett Syndrome

Prof. Adrian Bird Gives Speech at London Event

What Success Looks Like

Letter from the Executive Director

The X Factor

The Search for Novel Therapeutic Approaches for Rett Syndrome Broadens and Intensifies

A New Way of Doing Business

Rett Syndrome – A Disease of Neurons AND Glia

Bringing Justice to Rett

Rett Syndrome: Neurodevelopmental No Longer

Fragile X and Rett Syndrome – Opposite Ends of the Bell Curve?

The View Looks at Rett

Rett Syndrome In a Petri Dish

Yet Another Door Opens: Neuroimmunology

Curing Rett Syndrome: How do we get there? PART II

Scoliosis in Rett Syndrome

Redefining the Function of the Rett Syndrome Protein

Rett Syndrome and the DSM V

The Upside of Genetic Mutations

Rett Gene Discovery: Ten-Year Anniversary

Curing Rett Syndrome: How Do We Get There?

And Then There Were Six

RSRT Advisor Makes Significant Discovery