Rettsyndrome.org Blog Archives

 

Staying Healthy during the winter – A Focus on Your Lungs

December 19, 2016

Guest blogger Marianna M. Sockrider, MD, DrPH, Chief of Pulmonary Clinics Texas Children's Hospital shares how important it is to stay healthy  With the winter comes the cold and flu season.  Many people have respiratory infections during the winter months, in part because the cold weather and holiday season bring more people together inside.

50th Anniversary Rett Perspective

December 7, 2016

In September, we gather to commemorate Dr Andreas Rett on the 50th anniversary of his first publication describing this unique syndrome, to “look into the past towards a vision of the future”.  We owe so much to Dr Rett of Austria, and the handful of other keen observers like Dr Hagberg of Sweden and Dr Segawa of Japan for taking notice of our Silent Angels decades ago.  We celebrate them as professionals for speaking and writing about what we parents have always known in our hearts: 

A Case Study of Rettsyndrome.org's Scout Program

October 14, 2016

Rettsyndrome.org presents a case study of the successful partnership between our organization and a biopharmaceutical company (Anavex Life Sciences Corp.).  The study helps to identify potential new treatments for Rett syndrome.  This study also serves as an example of how a non-profit organization can encourage the pharmaceutical industry to test compounds and drugs.  These drugs are in their pipeline for preclinical testing and can support a non-profit organization.  In this case study, we cover the positive testing of the compound, ANAVEX 2-73, in a mouse model of Rett syndrome.  It also outlines Anavex’s plan to move ahead into a human clinical study.
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Power of data collected from the Natural History Study

October 5, 2016

Recently I had the pleasure to speak at the National Institute of Neurological Diseases and Stroke non-profit partners’ forum.  At the meeting I was asked to speak about the Rett syndrome (RTT) Research Consortium’s Natural History Study and the importance of the data collected in this study.  I would like to present a short synopsis of the origins of the Rett syndrome Natural History Study and the importance of it today as we move forward into more clinical trials.
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A Sibling’s Perspective of Rett Syndrome

September 1, 2016

Some people grow up with many siblings, some with a twin brother or sister, some as an only child. I grew up with two amazing younger sisters and a very special older sister. Her name is Paige and she has Rett syndrome.
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Expansion of the Rett syndrome pediatric trial with trofinetide (Neuren Pharmaceuticals)

August 25, 2016

The current multi-site pediatric clinical trial studying trofinetide has an enrollment rate that is running ahead of schedule, and to date, no safety or tolerability signals have been identified in this blinded trial.
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Visiting Your Child’s General Education Classroom

August 1, 2016

With the new school year starting soon, read more about children with Rett syndrome in the general education classroom.
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14th Rett Syndrome Research Symposium Outcomes

July 18, 2016

Rettsyndrome.org is pleased to announce we undoubtedly had another successful research symposium to encourage communication and collaboration within the Rett researcher community.
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Help Preserve Defense Research Dollars

June 3, 2016

Rettsyndrome.org is asking you to contact your Senator to ask her/him to help preserve DoD funding that may be used for research to aid our girls and our soldiers.
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Applying for Disability on Behalf of a Child with Rett Syndrome

May 25, 2016

Individuals with Rett syndrome qualify to receive disability benefits because they meet the Social Security Administration's medical eligibility requirements. Read on to find out how to apply and more about the benefits those with Rett syndrome can receive.
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Anavex 2-73 Receives the FDA's Orphan Drug Designation (ODD)

May 20, 2016

We recently told you about positive research results stemming from our collaboration with Anavex in the Rettsyndrome.org Scout Program.  We are now even more pleased to share the news that Anavex announced that their compound Anavex 2-73 has received the FDA’s Orphan Drug Designation (ODD). Read More »

Newron Pharmaceutical Announces Progress with Sarizotan

May 18, 2016

Newron Pharmaceutical just announced that their Investigational New Drug (IND) application for sarizotan has been approved.  Sarizotan is a novel compound for Rett syndrome, which had already been granted an Orphan Drug Designation. Read More »

Preparing for an Overnight Video EEG

May 2, 2016

Krysia Nelson, Mom to Kate along with Judy Barrish, RN, Blue Bird Circle Rett Center share ways to prepare for an overnight video EEG.  Read More »

Positive Data Released by Anavex Life Science Corps

February 25, 2016

Today Anavex Life Science Corps, a clinical-stage biopharmaceutical company and partner in Rettsyndrome.org’s Scout Program, just announced positive data of ANAVEX 2-73 in the Rett syndrome mouse model. Read More »

Foundation for the NIH Lurie Prize

February 16, 2016

Rettsyndrome.org is excited that Dr. Jeannie Lee is the winner of this year’s Lurie Prize from the Foundation for the NIH for outstanding work in epigenetics! Dr. Jeannie Lee was a previous grantee of Rettsyndrome.org , receiving a 2013 HeART award for the project “Treating Rett syndrome via selective reactivation of the silenced MECP2 allele”. Read More »

Our Research Approach

February 5, 2016

In 2015, tremendous progress was made in our efforts to advance Rett syndrome research. This year holds even more promise for Rett syndrome as Rettsyndrome.org continues to fund research programs. Continue reading to hear our approach for 2016. Read More »

Looking to Adulthood

January 19, 2016

A blog written by Judy O. Barrish, BSN, RN at The Blue Bird Circle Rett Center. Read More »

Worldwide Faces of Rett Syndrome: Russia May 2016 World Congress

January 8, 2016

In May 2016 Russia will host the World Congress on Rett syndome. Hear from one of the organizers of this event. Read More »

Rett Syndrome Now Eligible for $278 Million in Federal Research Dollars

December 30, 2015

In January of 2015, RSO (Rettsyndrome.org) undertook an ambitious advocacy campaign on Capitol Hill with the intent of enabling Rett syndrome research to be funded by the Department of Defense (DOD). Read More » 

New Details of the Pediatric Phase 2 Clinical Trial Study of Trofinetide

December 8, 2015

Neuren Pharmaceuticals is preparing a new Phase 2 clinical trial with trofinetide in a pediatric population with Rett syndrome. The study is aiming to enroll approximately 48 individuals, all of whom will receive trofinetide. The primary goal of the study will be to assess the tolerability and safety of the study medication in this younger population. Read More » 

Three Significant Trofinetide Announcements from Neuren Pharmaceuticals: An Overview

November 24, 2015

Neuren Pharmaceuticals has made three significant announcements in the last two months regarding trofinetide, the drug candidate they are developing as a treatment for Rett syndrome. Read More » 

A Parent's Guide to Flying with a Child with Rett Syndrome

August 7, 2015

Before you head out on your next trip read tips and tricks from other families on how to make your flight smooth. Read More » 

Neuren announces important progress in the US and Europe for trofinetide in Rett Syndrome

July 29, 2015

Neuren held a productive meeting with US Food and Drug Administration (FDA) about the drug trofinetide in treatment for Rett syndrome. Read More » 

Senate Appropriations Committee approves funding for research program with Rett Syndrome listed as an eligable condition

July 17, 2015

The Senate Appropriations Committee approves $278 for the Peer Review Medical Research Program and for the first time Rett syndrome is listed as a condition eligible for study in this program. Read More »

Newron's Sarizotan gets FDA orphan status to treat Rett Syndrome

July 15, 2015

Newron Pharmaceuticals has received orphan drug designation from the US Food and Drug Administration (FDA) for its new chemical entity (NCE) sarizotan to treat Rett syndrome. Read on to learn more about this new possible treatment option. Read More »

Gratitude in Every Step

July 9, 2015

Maria McEvoy is celebrating her birthday in a big way by climbing Mt. Kilimanjaro - the hightest mountain in Africa. She is doing it not only to accomplish a personal goal of hers but also to raise money for Rett syndrome. Read on to hear more about Maria's journey to Kilimanjaro. Read More » 

New Discovery About the X Chromosome Opens Up Possible Avenues for Future Treatments for Rett Syndrome

July 1, 2015

A critical paper regarding the X chromosome was published this week in Sciencexpress (June 18, 2015 pages 1-19) that will have a direct bearing on Rett syndrome in the foreseeable future. Read More »

Trofinetide for Rett Syndrome in Recent News

June 22, 2015

A novel agent is holding out some much-needed promise for girls and women with Rett syndrome. Read More »

Adaptive Bikes

May 20, 2015

Have you ever wondered what the benefits are of having an adaptive bike for your daughter or maybe where to get one? Read this blog post to learn all about adaptive bikes from several Rett parents. Read More »

Elected Officials are Considering Including Rett Syndrome Funding in the Department of Defense’s 2016 Budget

April 28, 2015

As the result of meetings held in Washington DC, many of our elected officials are thinking about Rett syndrome right now. Read More »

Revive Therapeutics Announces Positive Study Results From Its Research Collaboration With Rettsyndrome.org

April 28, 2015

Positive results announced by Revive Therapeutics as part of our Scout Program to identify potential new therapeutic drug treatments for Rett syndrome. Read More »

Surprising Contributor to Rett Syndrome Identified

April 21, 2015

Cells meant to maintain health worsening neurodevelopmental disorder's progression - University of Virginia Health System. Read More »

Neuren's Breakthrough Therapy Status Recent Update: What Does it Mean?

March 2, 2015

Today Neuren Pharmaceuticals announced that the U.S. Food and Drug Administration responded to their request for Breakthrough Therapy Status, one of several programs at the FDA for drug development. Read More » 

Today Neuren Pharmaceuticals announced that the U.S. Food and Drug Administration responded to their request for Breakthrough Therapy Status, one of several programs at the FDA for drug development.

Today Neuren Pharmaceuticals announced that the U.S. Food and Drug Administration responded to their request for Breakthrough Therapy Status, one of several programs at the FDA for drug development. - See more at: https://www.rettsyndrome.org/blog/neuren-breakthrough-status-explained#sthash.G3Lx43P5.dpuf

 

Today Neuren Pharmaceuticals announced that the U.S. Food and Drug Administration responded to their request for Breakthrough Therapy Status, one of several programs at the FDA for drug development. - See more at: https://www.rettsyndrome.org/blog/neuren-breakthrough-status-explained#sthash.G3Lx43P5.dpu

Neuren and the Orphan Drug Status: An Overview

February 17, 2015

This week’s news that Neuren Pharmaceuticals has been granted Orphan Drug Status by the FDA for NNZ-2566, now known as Trofinetide, is good news for families who live with Rett Syndrome. Read More » 

Accelerating Research: "We are in the Time of Rett"

February 11, 2015

We are living in an exciting and hopeful time - “the time of Rett” - where finding a treatment and a cure for Rett is truly possible. Read More » 

Be our Limited-Edition Designer! 

January 26, 2015

Rettsyndrome.org is looking for an artist! We have teamed up with Paper Clouds Apparel, a company that showcases the creative minds and abilities of individuals with special needs, to launch a limited edition design to raise funding for Rett. Read More » 

Revive Therapeutics Enters Into Research Collaboration With Rettsyndrome.org

January 15, 2015

TORONTO, ONTARIO--(Marketwired) - Revive Therapeutics Ltd. ("Revive" or the "Company") (TSX VENTURE:RVV) is pleased to announce that it has entered into a research collaboration with Rettsyndrome.org to explore the potential of Revive's REV-003 (Tianeptine) for the treatment of Rett syndrome.As part of the collaboration, Revive will supply the drug to conduct the study under the Rettsyndrome.org Scout Program. Read More »

NNZ- 2566 Clinical Trial: The Families Who Participated

December 23, 2014

Many of you know Geraldine (aka "Cake") from Virginia and her daughter Amanda.  We know her as a strong, capable, intelligent advocate and mother, who always has a smile on her face and a “can-do” attitude.  But there is more depth and detail to her life story than most of us know and she is willing to share a glimpse with us. Read More » 

"IEPs and School Placement: Exploring Perspectives Empowers the Parent” – a presentation for all school-age families

December 17, 2014

Don't sweat the small stuff . . . but an IEP really isn't small stuff. An IEP is HUGE! The IEP dictates our life at the school, and will influence your child's life after high school.  But . . . you probably already know how HUGE an IEP is.  So, why should you spend your extremely precious and limited time listening to this presentation?  Because it's powerful! Read More » 

How we choose a research project to fund and what exactly happens behind the scenes to ensure the research investments of Rettsyndrome.org are good ones.

December 15, 2014

We realize this blog is a comprehensive read, but it is also an extensive and vital subject. Understanding how we choose to fund Research Projects and what safeguards we have in place is as important to you as it is crucial to Rettsyndrome.org. Read More » 

NNZ-2566 success:  Breaking it down and answering one common question:  “What is this drug supposed to do for girls and women with Rett syndrome?”

November 13, 2014

It’s been an incredible week for the Rett syndrome community with the announcement from Neuron Pharmaceuticals about its successful phase 2 NNZ 2566 Clinical Trial. We wanted to take the time to answer one common question: “What is this drug supposed to do for girls and women with Rett syndrome?” Read More »

Current Clinical Trials in Rett Syndrome

November 13, 2014

In this blog I will cover the current status and progress of all drug intervention related Rett syndrome trials found on Clinicaltrials.gov. Read More »

Where Care Meets Cure: The Growth of Rett Clinics - UPDATE

November 7, 2014 (Update from August 4, 2014 post)

Rettsyndrome.org is committed to cultivating NETWORKED centers; our successes in this field means that we are regularly asked to consult on how to bring this clinic model to more cities in the U.S., and to more countries around the world. Read More »

The NIH Funded Natural History Study

November 5, 2014

Last week a co-operative agreement was announced to fund research for the Natural History Study, a Research Consortium, to be led by Dr. Alan Percy University of Alabama at Birmingham. Read More »

Curerett Gives Back

October 7, 2014

Curerett.org contributed an additional $150,000 to Rettsyndrome.org.  With this support, Rettsyndrome.org will be able to further push recent grants aimed at propelling translational research, launching the neuro-habilitation therapeutic program, and funding clinical research, which collective received $1.5M from Rettsyndrome.org. Read More »

October Rett Syndrome Awareness Month;
What Would You Give
?

September 30, 2014

Here we are on the brink of another October. For anyone who doesn't know, October is the month designated Rett Syndrome Awareness Month. That means every October, as a community of parents, families, friends and supporters, we do our best to raise the public consciousness of Rett syndrome. Read More »

Advancing Research.
Building programs to maximize our girls’ abilities (Neuro-Habilitation).

September 29, 2014

We’ve just announced some of the exciting studies in a brand new research area here at Rettsyndrome.org, and we wanted to take a moment to discuss what the program is and what aims to achieve. Read More »

Rettsyndrome.org Invests $1.5M for Translational Research and Establishes New Neuro-Habilitation Program for Rett Syndrome in the first half of 2014

September 19, 2014

The Rettsyndrome.org Board of Directors has awarded $1.5M to support 10 new grants to further translational research, launch the neuro-habilitation therapeutic program, and fund clinical research. Read More »

Where Care Meets Cure: The Growth of Rett Clinics & Centers in North America

August 4, 2014

By early 2015, Rettsyndrome.org will have helped inspire the development, growth and networking of 18 Rett specialty centers in North America, all passionately supporting our children and rapidly spreading the news that care and cure are linked hand in hand... Read More »

WELCOME Back a Leading Board Member

July 24, 2014

Rettsyndrome.org is proud to welcome long time Board of Directors member, Gordon (Gordy) Rich, back to the Rettsyndrome.org Board of Trustees. Gordy, with the support of his wife Anne, created the Kelly Rich Rett Research Foundation in 1997 for their daughter Kelly, who had just been diagnosed with Rett syndrome as well as funding... Read More »

Medical Marijuana in Neurologic Disease

May 5, 2014

A new review from the American Academy of Neurology (AAN) Review concerning Medical Marijuana in Neurologic Disease was published this week in the journal Neurology. For this systematic review, researchers looked at studies of marijuana in the treatment of symptoms of MS, epilepsy, and movement disorders reported from 1948 – November 2013... Read More »

A Human Clinical Trial Study on NNZ-2566

April 17, 2014

If you follow Rett research closely (or maybe even loosely), NNZ-2566 will be a drug name you’re familiar with. But if you're looking for information about this potentially groundbreaking new medication and the implications it may have for Rett syndrome, please read this Research Digest we developed with Cure Rett and Neuren to answer questions... Read More »

The Rett and Statins Paper Explained

April 16, 2014

You may have heard that "Statins Suppress Rett Symptoms in Mice". We know that title looks pretty bold and exciting. And it is! But possibly not for the reasons you think, and we are here to help break these things down in plain English... Read More »

Gene Therapy for Rett Syndrome?

March 2, 2014

As of late, there's been much buzz about gene therapy as a potential treatment for Rett syndrome. And for good reason! As many of us are aware of the pioneering 2007 reversal studies by Adrian Bird’s laboratory, it is hoped that by adding in a good copy of MECP2 through gene therapy methods could potentially improve symptoms of Rett syndrome or even potential reverse it... Read More »