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
Cannabidivarin preserved cognitive function and improved motor coordination in a female mouse model of Rett syndrome, a study suggests.| 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 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
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
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
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
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
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
Trofinetide significantly reduced neurobehavioral symptoms and improved communication in females with Rett syndrome, the LAVENDER trial shows.| 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
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
Anavex is preparing meetings with regulatory authorities in the U.S., Europe, and Asia-Pacific to discuss Anavex 2-73’s potential approval.| Alzheimer's News Today
Experimental blarcamesine (Anavex 2-73) was able to slow cognitive and functional decline in early Alzheimer’s patients over a year, data show.| Alzheimer's News Today