University, PharmaTher Join Forces to Develop Ketamine for Rett | Rett Syndrome News
Case Western Reserve University is partnering with PharmaTher to develop and commercialize ketamine for the treatment of Rett Syndrome.| Rett Syndrome News
No description.| Rett Syndrome News
Case Western Reserve University is partnering with PharmaTher to develop and commercialize ketamine for the treatment of Rett Syndrome.| Rett Syndrome News
About a month after its FDA approval, Daybue (trofinetide) is now available in the U.S. to treat Rett syndrome in patients 2 and older.| Rett Syndrome News
Ketamine, a dissociative anesthetic that makes users feel detached from their surroundings, is being studied clinically as a treatment for 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
Columnist Jacqueline Babiarz shares how her family promoted awareness about Rett syndrome by creating a Cammy Can superhero character.| Rett Syndrome News
At an Andrea Bocelli concert, columnist Jackie Babiarz and her daughter get a chance to meet the star backstage.| Rett Syndrome News
Category archive page for Columns.| Rett Syndrome News
After expatriation of a partnership agreement with Astellas, Taysha Gene Therapies regained full rights to TSHA-102, its experimental gene therapy being developed to treat Rett syndrome. “We are thrilled to regain full global rights to our lead TSHA-102 Rett syndrome program, which enables full strategic flexibility and optionality as we continue to advance the program […] The post Taysha regains full rights to TSHA-102 gene therapy for Rett appeared first on Rett Syndrome News.| Rett Syndrome News – The Web's Daily Resource for Rett Syndrome News
Columnist Jacqueline Babiarz's body continues to recover from the physical toll of caregiving two years after her daughter passed away.| Rett Syndrome News
NGN-401, a gene therapy treatment people with Rett syndrome, has shown positive results from an ongoing Phase 1/2 trial.| Rett Syndrome News
The Web's Daily Resource for Rett Syndrome News| Rett Syndrome News
A gene therapy showing potential to restore lost developmental milestones in people living with Rett syndrome has earned breakthrough therapy status from the U.S. Food and Drug Administration (FDA). The experimental therapy, TSHA-102, is being developed by Taysha Gene Therapies. A breakthrough therapy designation is intended to speed the development and review of important new treatments for […] The post FDA grants breakthrough status to Rett syndrome treatment appeared first on Rett Synd...| Rett Syndrome News – The Web's Daily Resource for Rett Syndrome News
October is Rett Syndrome Awareness Month, and supporters are going purple to bring attention to the rare genetic condition. “There’s no one right way to raise awareness,” the International Rett Syndrome Foundation (IRSF) says on its website. “Whether you’re posting on social media, sharing your story, or just starting a conversation, you’re helping others understand what […] The post Rett community goes purple in October for awareness month appeared first on Rett Syndrome News.| Rett Syndrome News – The Web's Daily Resource for Rett Syndrome News
Grief is complicated. There’s no playbook, but I wish there were. I try to prepare myself for events that I know will evoke grief, but I can’t stop it from bubbling up. My oldest daughter, Cammy, passed away in December 2023 at the age of 14 from complications of Rett syndrome. She had only been in […] The post A pink homecoming dress keeps sisters connected appeared first on Rett Syndrome News.| Rett Syndrome News – The Web's Daily Resource for Rett Syndrome News
Lab study findings on problems caused by MECP2 gene mutations in Rett syndrome may help scientists to better understand the rare disease.| Rett Syndrome News
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
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
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 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
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
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
Category archive page for News.| 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
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
