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Neurogene Announces Expansion of Gene Therapy Clinical Trial

Monday, March 4, 2024

Today, Neurogene announced plans to expand and more rapidly enroll patients their Phase 1/2 clinical trial of investigational gene therapy NGN-401. For the full press release, click here. Key Facts: The study will now expand to include a total of 16 pediatric participants (age 4-10). Cohort 1 (low dose group) will increase from 5 to … Read More

Taysha Gene Therapies Adolescent & Adult Study Expanded to U.S.

Thursday, February 29, 2024

Today, Taysha Gene Therapies shared several exciting updates on their REVEAL Phase 1/2 clinical trial of investigational gene therapy TSHA-102: The study will now expand to include adolescent and adult patients in the U.S. (age 12+ protocol). The dosing of the first cohort (low dose) is complete in the adult study in Canada with clinical … Read More

My Rett Ally: Revolutionizing Care Management for Rett Families

Thursday, January 11, 2024

On a recent visit to the emergency room, it took just seconds for Samantha Brant to pull up a detailed, up-to-date medication list for her daughter, Macy. Macy lives with Rett syndrome and Samantha, like all families with a loved one with Rett, must keep meticulous records of health documents to ensure the best possible … Read More

Acadia Pharmaceuticals Acquires International Rights to Trofinetide

Thursday, July 13, 2023

BIG NEWS for our international Rett syndrome community! Today, Acadia Pharmaceuticals announced they have acquired the rights to make trofinetide available for the treatment of Rett syndrome across the globe. READ THE PRESS RELEASE This is an essential starting point in ensuring ALL Rett families have access to this treatment. Acadia already retains the rights … Read More

RettAway Reflections

Tuesday, July 11, 2023

By Samantha Brant, Family & Community Engagement Manager, and mom to Macy As I returned home from this year’s RettAway at Morgan’s Inspiration Island, my heart swelled with not only pride but warmth. I attended the last RettAway in 2017 as simply a parent to a 3-year-old little peanut, Macy. And I took so much … Read More

IRSF Welcomes Three New Members to its Board of Directors

Monday, July 10, 2023

The International Rett Syndrome Foundation (IRSF), a mission-driven advocacy foundation focused on funding research and supporting families impacted by Rett syndrome, today announced the expansion of its Board of Directors. Mary Engel, Laura Stoppel, and Steve Wood have been appointed to the IRSF Board, effective July 1, 2023. “We are delighted to welcome these new … Read More

Clinical Trial Updates from Taysha & Anavex

Wednesday, June 28, 2023

We are excited to share news from two pharmaceutical companies working on potential treatments and cures for Rett syndrome. Taysha Gene Therapies Taysha Gene Therapies shared that they are encouraged by the initial clinical observations of the first adult patient dosed in their TSHA-102 REVEAL Phase 1/2 gene therapy trial in Canada. Further clinical updates … Read More

Neurogene Announces Locations & Opens Enrollment for US Gene Therapy Clinical Trial

Monday, June 12, 2023

We’re excited to share that today Neurogene announced the locations and began enrolling for the first U.S. Phase 1/2 clinical trial of their investigational gene therapy, NGN-401. Enrollment at Texas Children’s Hospital begins today, with enrollment at Children’s Hospital Colorado and Boston Children’s Hospital anticipated to open soon. The trial will enroll five (5) females aged … Read More

Taysha Announces First Patient Dosed with Gene Therapy

Monday, June 5, 2023

A First in Rett Syndrome! Taysha Gene Therapies announced today that the first Rett syndrome patient has been dosed with the investigational gene therapy, TSHA-102, in a clinical trial for females 18 and older being run out of Montreal, Canada. Read their press release and full community update below. ———————- June 5, 2023 Dear Rett … Read More

A First for Rett: FDA Approves Trofinetide for Treatment of Rett Syndrome!

Friday, March 10, 2023

This first-ever treatment is approved for individuals with Rett syndrome. (Updated March 19, 2023) On March 10, 2023, Acadia Pharmaceuticals announced that their investigational drug, Trofinetide, has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of Rett syndrome. The drug, marketed under the name DAYBUE™, is expected to be commercially … Read More

IRSF Establishes Research Fund for Males with Rett Syndrome

Tuesday, February 21, 2023

The fund is established thanks to an inaugural gift from the Otis Family in honor of their son, Barrett. The Rett syndrome research landscape has never been more exciting. New clinical trials are underway in gene therapy, and we eagerly await the FDA’s decision about Trofinetide. But we know that families of males with Rett … Read More

Taysha Gene Therapies Provides Update on Expansion of TSHA-102 Study

Wednesday, February 1, 2023

READ TAYSHA’S LETTER TO THE RETT COMMUNITY DALLAS, Jan. 31, 2023 (GLOBE NEWSWIRE) — Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, clinical -stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic rare diseases of the central nervous system (CNS), today provided an update on the TSHA-120 program in giant axonal … Read More

IRSF Awards $4.4M in Rett Syndrome Research Grants

Tuesday, January 31, 2023

Our commitment to setting up the next wave of Rett breakthroughs continues with the largest annual investment in our Foundation’s history. By Dr. Dominique Pichard, IRSF Chief Science Officer We are entering new territory for Rett syndrome. Right now, the FDA is evaluating the first-ever potential treatment specifically targeting Rett syndrome, trofinetide. No potential treatment … Read More

Neurogene Announces First Rett Syndrome Gene Therapy Trial in U.S.

Monday, January 23, 2023

We’re excited to share that Neurogene announced today that the FDA approved their Investigational New Drug Application (IND) to launch a clinical trial for their gene therapy treatment NGN-401 in 2023 in pediatric females. This is the second gene therapy clinical study in Rett syndrome currently underway and the very first gene therapy clinical trial … Read More

IRSF Receives $500,000 Donation from Vezbi Super App

Tuesday, January 3, 2023

International Rett Syndrome Foundation (IRSF) is proud to announce that it is the recipient of a $500,000 donation from the Vezbi Super App as part of its Project Seva. Along with the donation, Vezbi will be creating custom Micro-apps and Communities for use by IRSF and the community of families impacted by Rett syndrome that … Read More

Flu, COVID, & RSV: A Message to our Rett Community

Thursday, December 8, 2022

By Dr. Tim Benke, IRSF Medical Advisor As many of you know, the United States is currently facing the triple impact of flu, RSV, and COVID-19. These respiratory illnesses are currently circulating at levels higher than usual for this time of year, creating a surge of patients seeking care in clinical offices, emergency rooms, and … Read More

Taysha Begins Recruitment for Gene Therapy Trial

Thursday, November 17, 2022

Next Huge Milestone for Rett The last 12 months have been an unprecedented time for Rett syndrome. In December 2021, Acadia Pharmaceuticals reported that the first (and only) Phase 3 clinical trial in Rett syndrome had positive results. As hopefully many of you know, Acadia submitted a New Drug Application to the FDA for approval … Read More

Your Voice Matters to the FDA & Pharmaceutical Industry

Thursday, October 13, 2022

By Dr. Dominique Pichard, IRSF CSO – The patient voice has increasingly become a focus for the Food and Drug Administration (FDA) in the drug development process. Thanks to years of advocacy from patients and caregivers, the FDA has developed mechanisms to hear the patient voice and has encouraged pharmaceutical companies to increasingly include the … Read More

The 2022 Rett Gazette is Here!

Tuesday, October 11, 2022

Coming to your mailbox this October – our 2022 Rett Gazette is here! Find out how we are continuing to accelerate vital, innovative research while helping families thrive, no matter where they are on their Rett Journey. This year’s issue highlights how a world without Rett syndrome requires a powerful synergy between families and research, … Read More

FDA Grants Trofinetide Priority Review

Monday, September 12, 2022

ACADIA has shared the exciting news that the US Food and Drug Administration (FDA) has reviewed the documents received as part of the New Drug Application (NDA) for trofinetide, and they have determined that the application is complete and can be filed. They also determined that trofinetide qualifies for Priority Review. The Prescription Drug User … Read More

In-Person Strollathons Are Back!

Tuesday, July 26, 2022

In September IRSF returns with our in-person Strollathons! These nationwide fundraisers bring together local communities to go ALL IN for treatments and a cure for Rett syndrome. Since 2004, these family-friendly events powered by dedicated volunteers, sponsors, donors, and Rett families have raised more than $15M to advance IRSF’s mission to empower families and accelerate … Read More

IRSF Expands Center of Excellence Network

Wednesday, July 20, 2022

The International Rett Syndrome Foundation is expanding our community’s access to best-in-class care by adding four Rett syndrome clinics to our Center of Excellence network. This expansion helps to fulfill IRSF’s commitment that all families living with Rett syndrome in the U.S. have ready access to a Rett syndrome expert. “When we see care deserts, … Read More

Acadia Community Update on Trofinetide

Wednesday, July 20, 2022

July 18, 2022 Dear Rett Community, Acadia is pleased to announce the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for trofinetide for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older. The full text of the Acadia press release can … Read More

The Summit of Hope and Healing: ASCEND 2022 Rett Syndrome National Summit

Wednesday, June 8, 2022

By Katie Busch, IRSF Program Coordinator –  During the last week of April 2022, the International Rett Syndrome Foundation welcomed more than 500 members of the Rett community to the ASCEND 2022 Rett Syndrome National Summit. Held in Nashville, Tennessee, at the Gaylord Opryland Resort & Convention Center, the sold-out summit marked the first in-person … Read More

Clinical Trial Committee

IRSF Launches Clinical Trial Committee to Support the Development of Rett Syndrome Treatments

Tuesday, May 31, 2022

The Clinical Trial Committee includes 14 leading U.S. Rett syndrome specialists CINCINNATI – (May 31, 2022) – The International Rett Syndrome Foundation (IRSF) announced today that its Clinical Trial Committee (CTC) has launched, having completed its first protocol review, and is available as a resource to drug developers and pharmaceutical companies planning clinical trials to … Read More

Accelerating Discoveries in Rett Research: 2022 IRSF Rett Syndrome Scientific Meeting

Thursday, May 26, 2022

In April 2022, the International Rett Syndrome Foundation hosted the IRSF Rett Syndrome Scientific Meeting in advance of the foundation’s first in-person family summit in 6 years. The 2-day meeting brought together more than 150 clinical, academic, and industry experts from around the world, all working towards the common goal of developing effective treatments and … Read More

Taysha Rett Syndrome Community Update

Wednesday, March 30, 2022

From IRSF: 2022 is a big year for Rett syndrome: the return of an in-person Family Summit, a successful EL-PFDD meeting to share the voice of the community with the FDA, ACADIA will submit a new drug application to the FDA to request approval of trofinetide as the first medication specifically targeting Rett syndrome, and … Read More

Federal Funding Bill for 2022 Supports Research for Rett Syndrome

Monday, March 28, 2022

CINCINNATI – March 22, 2022 – The Omnibus Fiscal Year 2022 (FY22) appropriations bill passed by Congress in early March includes federal funding to support research on Rett syndrome. The report accompanying the legislation listed Rett syndrome as a topic eligible for research funding through the U.S. Department of Defense (DoD) Peer-Reviewed Medical Research Program … Read More

2021 IRSF funded research

IRSF Sets Up Next Wave of Rett Breakthroughs with $2.3 M Research Investment

Wednesday, December 22, 2021

Dr. Dominique Pichard, IRSF Chief Science Officer Get ready for the next wave of research targeting treatments and cures for Rett syndrome. IRSF is making it happen. “Because Rett syndrome is a rare disorder, our Foundation’s investments strongly influence the direction and pace of Rett research, as well as the potential treatment options that come … Read More

Positive Trofinetide Results

Positive Top-line Results for Phase 3 Trofinetide Trial

Monday, December 20, 2021

Melissa Kennedy, Chief Executive Officer On December 6, the Rett syndrome community received some very encouraging news: Acadia Pharmaceuticals reported that top-line results for its Lavender Phase 3 clinical trial for trofinetide showed significant improvement over placebo. With these positive findings, Acadia will request a pre-New Drug Application meeting with the FDA in the first quarter … Read More

COVID UPDATE: Best practices according to our medical expert

Friday, December 10, 2021

As the COVID pandemic continues with surges and new variants, we have asked IRSF Medical Advisor Dr. Tim Benke, Rett Clinic Medical Director at Children’s Hospital Colorado, to provide information related to COVID-19 to keep our families and community healthy and safe at this time. Vaccinations: We continue to advise: unless told otherwise by their … Read More

Acadia Pharmaceuticals Announces Positive Top-line Results from the Pivotal Phase 3 Lavender Trial of Trofinetide in Rett Syndrome

Monday, December 6, 2021

– Trofinetide met co-primary efficacy endpoints demonstrating statistically significant improvement over placebo in the Rett Syndrome Behaviour Questionnaire (RSBQ) (p=0.0175) and the Clinical Global Impression of Improvement (CGI-I) (p=0.0030) – Trofinetide met key secondary endpoint demonstrating statistically significant improvement over placebo in CSBS-DP-IT–Social (p=0.0064), caregiver scale of ability to communicate – Pre-New Drug Application meeting … Read More

Hive Networks & IRSF Partner to Advance Research and Treatment for Rett Syndrome

Monday, October 18, 2021

Hive Networks today announced a multi-year partnership with the International Rett Syndrome Foundation to provide a digital health platform to support the Rett community. Rett syndrome is a rare disease caused by a genetic mutation that affects 1 in 10,000  females and even fewer males.   Hive Networks is a digital health company that lets patients … Read More

IRSF Appointed to NHC Standard of Excellence Program

Friday, September 10, 2021

The National Health Council (NHC) Standard of Excellence program recently appointed IRSF to its prestigious membership. The NHC is a 100-year-old organization whose mission is to provide a united voice for the 160 million people living with chronic diseases and disabilities. Created by and for patient organizations, NHC envisions a society in which all people … Read More

COVID UPDATE: Best practices according to our medical expert

Tuesday, August 17, 2021

As COVID numbers surge once again, we have asked IRSF Medical Advisor Dr. Tim Benke, Rett Clinic Medical Director at Children’s Hospital Colorado, to provide information related to COVID-19 to keep our families and community healthy and safe at this time. Vaccinations: We continue to advise: unless told otherwise by their personal physician, all individuals … Read More

Help Unlock Treatments & Cures for Rett with IRSF’s Rett Syndrome Registry

Thursday, June 17, 2021

Your family can be the key that researchers and industry need to create a world without Rett. And the International Rett Syndrome Foundation (IRSF) is making it easier than ever for families to help unlock treatments and cures through the new Rett Syndrome Registry™. “Everything IRSF does is designed to accelerate the creation of treatments … Read More

International Rett Syndrome Foundation’s New Center of Excellence Network

Tuesday, May 4, 2021

The International Rett Syndrome Foundation is committed to advancing and expanding our community’s access to best-in-class Rett syndrome care. Toward that end, we are designating 15 partner clinics across the U.S. to our Center of Excellence Network. “Establishing this network is part of IRSF’s goal to get families the expert Rett clinical care that their … Read More

COVID Update: Rett syndrome and the COVID vaccine

Monday, January 25, 2021

1.25.21 COVID UPDATE: Best practices according to our medical expert. Last month, we shared our recommendations from the Rett Medical Community regarding the Covid-19 vaccine: unless told otherwise by their personal physician, all individuals with Rett syndrome and their families, caregivers, therapists, and teachers should be vaccinated. We are awaiting approval for those younger than 16 for … Read More

Kelsey & Toyua: Friendship & Hope Just 20 Minutes Away

Wednesday, November 18, 2020

Kelsey Clark felt alone and isolated when her five-year-old daughter Charlee was diagnosed with Rett syndrome in April 2019. Kelsey, her partner Jordan, Charlee, and their two-year-old son Bennett live just over an hour from New Orleans in the small town of Amite—population 4,200—and had never even heard of the disease before. Kelsey’s grandmother, a … Read More

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, International Rett Syndrome Foundation has partnered … 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

IRSF Announces New Scientific Advisory Board

Thursday, April 30, 2020

Exceptional Group of Scientific Leaders to Guide Foundation’s Research Investment As IRSF’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 International Rett Syndrome Foundation 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 … 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

Taysha Gene Therapies Doses First Pediatric Patient in U.S. Gene Therapy Clinical Trial

Wednesday, January 10, 2024

Taysha Gene Therapies today announced the dosing of the first pediatric patient in their REVEAL Phase 1/2 trial of investigational gene therapy TSHA-102. For Taysha’s full press release, click here. Key Facts: The first female pediatric patient was dosed at RUSH University Medical Center in Chicago, an IRSF-designated Center of Excellence. The REVEAL Phase 1/2 … Read More

Neurogene Announces First Pediatric Patients Dosed in US Gene Therapy Clinical Trial

Thursday, November 30, 2023

We’re excited to share that today Neurogene announced that two pediatric patients have been dosed in their Phase 1/2 trial of investigational gene therapy NGN-401. For Neurogene’s full press release, click here. Key Facts: The first two female pediatric patients were dosed sequentially in the 3rd and 4th quarter of this year at Texas Children’s … Read More