Cannabidivarin aids with motor skills, cognition in female Rett mice
Cannabidivarin preserved cognitive function and improved motor coordination in a female mouse model of Rett syndrome, a study suggests.| 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
NTI164, a cannabis-based oil being developed by Neurotech International, may help girls with Rett syndrome communicate more easily, enjoy better moods, and have a better quality of life with few side effects, a small study in Australia found. The study, “Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) Improves Symptoms of Rett Syndrome: An Open-Label […] The post Cannabis-based oil eases symptoms in Rett syndrome girls appeared first on Rett Syndrome News.| Rett Syndrome News – The Web's Daily Resource for Rett Syndrome News
Getting my hair washed at a salon is one of my guilty pleasures. It feels like I’m being pampered, and the deep scalp massage feels like a luxury. But last week, as my head was in the sink, I felt tears welling up. I closed my eyes to try to stop it, but the teardrops […] The post Something seemingly simple created a special bond with my late daughter appeared first on Rett Syndrome News.| Rett Syndrome News – The Web's Daily Resource for 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 or misconceptions in the medical community about Rett syndrome occurring in boys and men commonly leads to misdiagnoses or diagnostic delays, according to a new study. “Diagnostic providers must increase their awareness of males with [Rett] and be prepared to deliver the diagnosis with empathy and accurate, up-to-date information on prognosis […] The post Lack of awareness spurs diagnostic delays for Rett syndrome in males appeared first on Rett Syndrome News.| Rett Syndrome News – The Web's Daily Resource for Rett Syndrome News
Many types of movement disorders such as uncontrolled muscle contractions and Parkinson’s-like motor changes often co-occur with Rett syndrome, a study shows. The severity of movement disorders is associated with Rett’s overall severity, with uncontrolled muscle contractions particularly linked with breathing trouble and digestive problems, data indicate. The findings show the importance of systematically evaluating […] The post Different types of motor disorders often co-occur in Rett ...| Rett Syndrome News – The Web's Daily Resource for 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
Unravel Biosciences' agreement with Quality Chemical Laboratories will work toward manufacturing materials for clinical trials of RVL001.| Rett Syndrome News
Acadia is seeking regulatory approval in the European Union of trofinetide, sold in the U.S. as Daybue, for patients with Rett syndrome.| Rett Syndrome News
Steve Bryson is a science writer for Rett Syndrome News with a PhD in biochemistry. He covers the latest news and information on a variety of Rett syndrome topics.| Rett Syndrome News
The brain’s electrical activity in response to sound may serve as a biomarker for Rett treatment response, per a study.| Rett Syndrome News
Taysha Gene Therapies announced details for the second part of a clinical trial testing Rett gene therapy TSHA-102.| 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
