Recent Blogs

Find stories from other families and employees for Rettsyndrome.org who are willing to share their wisdom, experiences, worry, and hope with you! Know that you are not alone in this journey and become a part of a community who is making strides towards finding a cure.

Setting the Standard in Best-in-Class Care and Rett Syndrome Centers of Excellence

Thursday, September 17, 2020

RSO Establishes Medical Advisory Board to Enhance Clinical Care and Research Availability Creating a world without Rett syndrome requires a national network of clinics that are working closely together, sharing best practices, ensuring excellent care, and standing ready to participate in clinical trials. To make this network a reality, Rettsyndrome.org has partnered with 11 esteemed clinical … Read More

Serving & Equipping Families with Rett

Wednesday, July 29, 2020

By Samantha Brant, Family & Community Engagement Manager, Mom to Macy When most families find out their child has Rett syndrome, it’s the first time they’ve ever heard of the disease. Often they’re overwhelmed with emotions and fear and questions as they try to figure out what to do next, and many families search the … Read More

Gene Modifying Therapy

Gene Modifying Therapy Research: Accelerating Toward a Cure

Wednesday, July 22, 2020

By Dominique Pichard, MD, Chief Science Officer, Rettsyndrome.org We wanted to explain this incredibly complicated work and at the same time let you know that we’ve got you covered. We are working hard to provide a better life for your loved ones with Rett syndrome. It is believed that the greatest life improvements will come … Read More

Rettsyndrome.org Launches Rett Research Ready™ and myRett Trial Finder

Wednesday, July 15, 2020

Program supports participation in clinical trials to develop treatments for the rare condition Rettsyndrome.org has launched a program to enable families with loved ones affected by Rett syndrome to explore and participate in clinical research. Rett Research Ready™ is an online platform that includes education, resources and support to help families consider volunteering for research … Read More

Board of Directors Welcomes Alan Shukovsky —The Father of a Boy Living With Rett

Friday, July 10, 2020

When Alan Shukovsky and his wife, Eva, were getting married, their rabbi told them that it was really important for their family to find a personal mission—a cause—they could support throughout their lives. Alan and Eva Shukovsky welcomed their son Grayson on April 2, 2015, but at around six months they became concerned about his … Read More

COVID-19 Message for the Rett Community

Thursday, June 25, 2020

6.25.20 COVID-19 Community Update As many states begin to re-open, we want to share the following message containing the re-opening status of the U.S. Rett Clinics and guidance from Rettsyndrome.org’s Medical Advisor on returning to therapies and schools. Rett Clinic Re-opening Status The following information summarizes U.S. clinics’ re-opening status for clinical care of Rett … Read More

Rettsyndrome.org Announces New Scientific Advisory Board

Thursday, April 30, 2020

Exceptional Group of Scientific Leaders to Guide Foundation’s Research Investment As Rettsyndrome.org’s Chief Science Officer, a physician, and a mom to a teenager with Rett syndrome, I am dedicated to overcoming the challenges presented by Rett syndrome’s complexity on the research front. Recognizing that the best way to do this is to have input from … Read More

Child looking at Tobii

NEW Resources to Empower & Equip You During COVID-19

Friday, April 17, 2020

Rettsyndrome.org has just released these resources to support families who are confined to their homes We know that the shelter-in-place guidelines are especially challenging for our families caring for a loved one with Rett syndrome. Many therapies have been stopped or are being done in an abbreviated version so that gained ground is not lost. … Read More

Chief Science Officer Dr. Dominique Pichard

A Note from Chief Science Officer Dr. Dominique Pichard

Tuesday, November 12, 2019

A Note from Chief Science Officer Dr. Dominique Pichard I am thrilled to be joining Rettsyndrome.org as the Chief Science Officer. Rett syndrome is near and dear to me and my family, as our oldest daughter, Catalina, has Rett syndrome. We received Catalina’s diagnosis when I was still in my medical residency training, and ultimately … Read More

Prichard family

When Molly met Jill: Rett Connection Becomes Fast Friendship

Friday, November 8, 2019

— Becky Sher Molly Prichard jokes that she and best friend Jill Black met “online dating.” And to hear them talk about their fast friendship does sound like a conversation about Internet matchmaking — they mention instant connections, plans to meet in person after finding each other online, and even family members’ concerns that the … Read More

Collage of rett patients

Unwavering in Our Mission

Thursday, October 31, 2019

Rett Syndrome Awareness Month has been incredible. As a community, you rallied like never before – speaking out to share the good, the bad, and the ugly about Rett syndrome to educate the world and move the needle. Your efforts continue to make a difference. Today, more people know about Rett syndrome than ever before … Read More

Acadia pharmaceutials

ACADIA Announces Launch of Phase 3 LAVENDER Study for Trofinetide

Wednesday, October 30, 2019

Dear Rett Community, Today we took a significant step forward with the trofinetide clinical development program initiating the pivotal Phase 3 LAVENDER study. As we have shared before, LAVENDER is a 12-week study that will evaluate the efficacy and safety of trofinetide versus placebo in approximately 180 girls and young women aged 5 to 20 … Read More

Dr. Dominique Pichard

Rettsyndrome.org Announces New Chief Science Officer with Rett Syndrome Connection

Tuesday, September 17, 2019

The International Rett Syndrome Foundation (doing business as Rettsyndrome.org) has officially welcomed Dominique Pichard, MD to the organization as their new Chief Science Officer. A highly accomplished clinician and researcher, Dr. Dominique Pichard brings years of hands-on experience conducting critical work in clinical trials with the National Institutes of Health (NIH), pharmaceutical companies, and the … Read More

Jeannie T. Lee, MD, Ph.D and Davut Pehlivan, MD

New Research Funding to Provide Hope to Girls and Boys

Tuesday, June 4, 2019

Rettsyndrome.org Funds Research to Provide Hope for Females and Males Affected by MECP2 Mutation. CINCINNATI, OH – June 3 – Rettsyndrome.org announced their funding of two new research projects today. Jeannie T. Lee, MD, Ph.D. of Massachusetts General Hospital is awarded a two year ANGEL Grant for $600,000 to focus on reactivating the silent X … Read More

Learning Collaborative Group Shot

RSO Hosts Learning Collaborative for Rett Clinics

Tuesday, June 4, 2019

Rettsyndrome.org hosted a clinical Learning Collaborative on May 8 and 9 in Denver. The Learning Collaborative is made up of physicians and clinicians from Rett clinics around the country who volunteered their time to share ideas in the spirit of advancing care for Rett families, and to create a universal standard of excellence across all … Read More

Dr. Horrigan

Rettsyndrome.org Appoints Joseph Horrigan, MD to Board of Directors

Friday, May 3, 2019

Rettsyndrome.org Appoints Joseph Horrigan, MD to Board of Directors CINCINNATI, OH – May 3 – Rettsyndrome.org is pleased to announce that Joseph P. Horrigan, MD has joined its Board of Directors. Dr. Horrigan’s appointment follows the excitement of several research and clinical trial advancements supported or developed through Rettsyndrome.org. Dr. Horrigan is a pediatric neuropsychiatrist … Read More

Little girl with rettsyndrome

You are not alone

Monday, April 29, 2019

Did your child recently receive a Rett syndrome diagnosis? You are not alone. Rettsyndrome.org meets you at diagnosis and helps you through every facet of this unexpected journey. Our mission is to accelerate research toward treatment and a cure, but we are also committed to empowering families. It’s essential that families are equipped with information, … Read More

Positive Phase 2 Study Results of Trofinetide in Pediatric Rett Syndrome Published in Neurology

Wednesday, March 27, 2019

Positive Phase 2 Study Results of Trofinetide in Pediatric Rett Syndrome Published in Neurology, the Medical Journal of the American Academy of Neurology — Statistically Significant Improvement Indicates Trofinetide’s Potential for Treating Rett Syndrome — Trofinetide for Rett Syndrome has Fast Track Status and Orphan Drug Designation in the U.S. and Orphan Drug Designation in … Read More

Dr. Steven Kaminsky

Dr. Steven Kaminsky Retiring, RSO Honors Chief Science Officer

Thursday, March 7, 2019

Dr. Steven Kaminsky Retiring, RSO Honors Chief Science Officer Chief Science Officer Dr. Steven Kaminsky has announced his retirement this spring. Steve has served faithfully for the last seven years advancing research toward the treatment and cure of Rett syndrome. Instrumental in connecting trofinetide – a compound developed under the Department of Defense to treat … Read More

Couple with child

Connecting Families to Research

Tuesday, February 12, 2019

Connecting Families to Research From the beginning, Rettsyndrome.org (RSO) has been dedicated to accelerating research for the treatment and cure of Rett syndrome. We are equally dedicated to connecting families with information and tools to persevere through the many challenges they face while they wait for a cure. The generous support of our families and … Read More

Our Family’s Experience Participating in the Natural History Study

Thursday, April 26, 2018

Samantha Brant, Family & Community Engagement Manager, Rettsyndrome.org April 26, 2018 When my daughter, Macy, was diagnosed with Rett syndrome, we entered a new and often confusing world. There was so much to learn and adjust to. I dealt with “Why Me” and “Why Her” as I struggled to understand why my daughter had to … Read More

Pioneers Add to RSO’s Value in Clinical Trial Process

Monday, April 23, 2018

Paige Nues, Director of Family Empowerment Rettsyndrome.org In a world of complexity, I aim for simplicity. My work at Rettsyndrome.org has always been to focus around the age-old basics: Why, What, Where and When. My ‘Why’ started simply: our daughter Katie was diagnosed with Rett syndrome and I couldn’t sit by and wait for someone … Read More

Implications of Clinical Trials: A View of the Panorama

Tuesday, March 20, 2018

Alan Percy, MD and Walter Kaufmann, MD. The advances in basic and clinical research over the past two decades since the identification of mutations in the gene for most individuals with Rett syndrome (RTT) has been astonishing. Within the past few years, we have seen the number of clinical trials involving potential disease-modifying pharmaceutical agents … Read More

Our Research Delivers

Wednesday, March 14, 2018

Having recently joined Rettsyndrome.org, I am humbled and grateful for the opportunity to serve this very special community. My 30 years as a health care administrator has taught me that medical solutions to even the simplest condition can be complicated and challenging. For example, there are about 75 drugs to treat something as “simple” as … Read More

COVID Update: Advice Regarding Returning to School

Tuesday, August 11, 2020

By Dr. Tim Benke, Rettsyndrome.org Medical Advisor While we do not have any specific data, such as reports in the literature, children with Rett syndrome may be at high risk of complications due to Covid-19.  Children with Rett syndrome are prone to stereotypies in which they constantly bring their hands to their mouths.  Not only … Read More