One year of daily Daybue (trofinetide) treatment led to improvements in behaviors such as nonverbal communication, alertness, and social interaction and connectedness among children and adults with Rett syndrome. That’s according to published interim data from Acadia Pharmaceuticals‘ Phase 4 LOTUS study that assessed the safety and effectiveness of the approved oral therapy in 227 […] The post Daybue shows real-world benefits for Rett syndrome patients: Study appeared first on Rett Synd...| Rett Syndrome News – The Web's Daily Resource for Rett Syndrome News
Most people from my parents’ generation remember where they were when President John F. Kennedy was assassinated in 1963. Sept. 11, 2001, was my generation’s “Where were you when …?” I was driving to an 8 a.m. class when I heard on the radio that a plane had crashed into the World Trade Center. Classes […] The post In events of mass violence, who protects people with disabilities? appeared first on Rett Syndrome News.| Rett Syndrome News – The Web's Daily Resource for Rett Syndrome News
Add-on treatment with oral cannabidiol, known as CBD, helped lower seizure frequency in two-thirds of children with Rett in an Italian study.| Rett Syndrome News
Girls with Rett syndrome often face challenges when it comes to eating and drinking, according to a 20-year analysis. Due to limited hand function and difficulties with chewing and swallowing, many are unable to feed themselves or handle a wide variety of foods and liquids. As these girls get older, they typically continue to need […] The post Feeding challenges for girls with Rett syndrome explored in study appeared first on Rett Syndrome News.| Rett Syndrome News – The Web's Daily Resource for Rett Syndrome News
Last week, I attended a Crisis Prevention Institute (CPI) training session at the school where I work, and I couldn’t stop thinking about my late daughter, Cammy. Listening to examples of students’ trauma and precipitating factors that staff and fellow classmates might not be aware of reminded me of my own child’s situation. Educating our […] The post Crisis prevention training reminded me of my late daughter appeared first on Rett Syndrome News.| Rett Syndrome News – The Web's Daily Resource for Rett Syndrome News
Reduced production of orexin, a signaling molecule, is associated with problems in sleep-wake cycles in Rett syndrome mice, a study found.| Rett Syndrome News
Learn more about scoliosis, an abnormal curvature of the spine, and the most common orthopedic complication in Rett syndrome patients.| Rett Syndrome News
Blocking a small RNA molecule that helps control whether certain genes are switched on or off reactivated the healthy copy of the MECP2 gene in a mouse model of Rett syndrome, increasing lifespan and activity, and improving breathing. While blocking miR‑106a appears to be “a feasible therapeutic strategy,” before it can be advanced to clinical […] The post Gene therapy ‘sponge’ revives healthy gene in Rett syndrome appeared first on Rett Syndrome News.| Rett Syndrome News – The Web's Daily Resource for Rett Syndrome News
I’m the youngest of six children: four girls, a boy, then another girl. (That last would be me.) I was always jealous that my sisters were able to be together while attending the same high school and college. Hearing their memories of carpooling, hanging out, being teammates, and preparing for dances sounded so fun. There’s […] The post Preparing our youngest for high school is not what we’d hoped appeared first on Rett Syndrome News.| Rett Syndrome News – The Web's Daily Resource for Rett Syndrome News
Vorinostat, an approved lymphoma treatment sold as Zolinza, was found to ease both neurological and non-neurological symptoms in a mouse model of Rett syndrome in a new study. When an oral formulation of vorinostat was administered after symptom onset, the drug was even more effective than Daybue (trofinetide), the only approved treatment for Rett, which […] The post Use of cancer drug vorinostat shown to ease symptoms in Rett mice appeared first on Rett Syndrome News.| Rett Syndrome News – The Web's Daily Resource for Rett Syndrome News
Cannabidivarin preserved cognitive function and improved motor coordination in a female mouse model of Rett syndrome, a study suggests.| Rett Syndrome News
Newron Pharmaceuticals stopped work on sarizotan in May 2020 after it failed to show superiority to placebo in treating breathing problems in Rett patients.| Rett Syndrome News
Learn about common symptoms of Rett syndrome, which can range from gradual slower development to loss of motor and communication skills to seizures.| Rett Syndrome News
The Clinical Severity Scale (CSS) includes 13 items, with a total score ranging from 0 to 58, higher scores indicating more disease severity.| Rett Syndrome News
Treatment with esketamine, a compound related to ketamine, led to some behavioral improvements in three girls with Rett syndrome in a trial.| Rett Syndrome News
Columnist Jacqueline Babiarz illustrates how her daughter's diagnosis led her to denial, anger, bargaining, depression, and acceptance.| Rett Syndrome News
Columnist Jackie Babiarz shares how her family and friends came together on March 10 to celebrate her daughter Cammy's heavenly birthday.| Rett Syndrome News
Columnist Jacqueline Babiarz reflects on the passing of her 14-year-old daughter, Cammy, due to complications from Rett syndrome.| Rett Syndrome News
Lindsey Shapiro is a science writer for Rett Syndrome News with a PhD in neuroscience. She covers the latest news and information on a variety of Rett syndrome topics.| Rett Syndrome News
A lack of awareness in the medical community about Rett syndrome in males commonly leads to misdiagnoses or diagnostic delays, a study found.| Rett Syndrome News
Neurogene received clearance from the FDA for a pivotal trial of NGN-401, which could support an application for the gene therapy's approval.| Rett Syndrome News
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Columnist Jacqueline Babiarz's daughter with Rett syndrome had to endure a long stay in the pediatric ICU due to pneumonia and COVID-19.| Rett Syndrome News
In the study, lower bone mineral density was tied to small size, impaired mobility, and the use of valproic acid, an anti-seizure medication.| Rett Syndrome News
Pending regulatory clearance in Colombia, Unravel Biosciences will launch a trial this summer testing Rett treatment RVL001 in 15 patients.| Rett Syndrome News
NLX-101 improves breathing issues and cognitive function in a female mouse model of Rett syndrome, a study showed.| Rett Syndrome News
Columnist Jacqueline Babiarz offers a birthday tribute and explains the complexities of parenting siblings of a child with special needs.| Rett Syndrome News
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Amid the depression and grief she feels in winter, columnist Jackie Babiarz finds something to boost her spirits: balloons.| Rett Syndrome News
On the anniversary of her oldest daughter's death, columnist Jacqueline Babiarz has cried herself to sleep remembering those last 24 hours.| Rett Syndrome News
Columnist Jacquelyn Babiarz senses that her daughter's angel wings helped a supporter fly across the finish line in record time.| Rett Syndrome News
The first four Rett syndrome patients treated with the gene therapy candidate NGN-401 have seen meaningful gains in skills, data show.| Rett Syndrome News
Low-dose treatment with shock wave therapy for three months was seen to ease spasticity and improve motor function in Rett syndrome children.| Rett Syndrome News
NGN-401 is one of six picked, three each by the Center for Biologics Evaluation and Research, and the Center for Drug Evaluation and Research.| Rett Syndrome News
Patricia Inacio is a science writer for Rett Syndrome News with a PhD in cell biology. She covers the latest news and information on a variety of Rett syndrome topics.| Rett Syndrome News
A brain surgery called corpus callosotomy reduced seizure burden in a young girl with Rett syndrome and hard-to-treat seizures, per a report.| Rett Syndrome News
In light of Rare Disease Day 2022, columnist Jacqueline Babiarz, who has a daughter with Rett syndrome, considers what it means to be rare.| Rett Syndrome News
Learn more about how Rett syndrome is diagnosed, using physical examination, a review of medical history, and genetic testing.| Rett Syndrome News
Approved in the U.S. in March 2023, Daybue (trofinetide) is a daily treatment for Rett syndrome that can be taken by mouth or feeding tube.| Rett Syndrome News
Andrea Lobo is a science writer for Rett Syndrome News with a PhD in cell biology/neurosciences. She covers the latest news and information on a variety of Rett syndrome topics.| Rett Syndrome News
Trofinetide significantly reduced neurobehavioral symptoms and improved communication in females with Rett syndrome, the LAVENDER trial shows.| Rett Syndrome News
Marisa Wexler is a senior science writer for Rett Syndrome News with an MS in cellular and molecular pathology. She covers the latest news and information on a variety of Rett syndrome topics.| Rett Syndrome News
When healthcare providers go above and beyond, they deserve our recognition, writes columnist Jacqueline Babiarz.| Rett Syndrome News
Columnist Jacqueline Babiarz explains how sharing photos of her late daughter's life with Rett syndrome helped her and countless others.| Rett Syndrome News
Learn more about what causes Rett syndrome, a rare genetic disorder that mostly affects females.| Rett Syndrome News
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The aftermath of losing a child is full of difficult firsts. Columnist Jacqueline Babiarz faces a tough back-to-school month.| Rett Syndrome News
The US regulatory agency acts to support the potential gene therapy's development, with ongoing Phase 1/2 trial showing safety in patients.| Rett Syndrome News
Daybue last year became the first Rett syndrome treatment to win U.S. Food and Drug Administration approval, and is up for approval in Canada.| Rett Syndrome News
ADH-503, a possible cancer therapy affecting immune cell activity, slowed progression and prolonged survival in a Rett syndrome mouse model.| Rett Syndrome News
Long-term treatment with Daybue (trofinetide) led to continual improvements in symptoms in people with Rett syndrome in extension studies, data show.| Rett Syndrome News
The International Rett Syndrome Foundation designated three new centers of excellence for clinical care of Rett patients.| Rett Syndrome News
A survey of clinicians found parental and familial support the biggest factors in favoring invasive ventilation for teens with Rett syndrome.| Rett Syndrome News
Work in a Rett cell model found the potential gold nanocrystal therapy significantly improved mitochondrial function and neuronal health.| Rett Syndrome News
Learn more about the rare neurodevelopmental disorder Rett syndrome, including its causes, symptoms, diagnosis and treatment.| Rett Syndrome News