Taysha Gene Therapies is partnering with Astellas Pharma to support the development of its gene therapy programs for Rett syndrome and giant axonal neuropathy, another progressive neurodegenerative disorder. Astellas will invest $50 million to acquire 15% of Taysha and to receive an exclusive option to license Taysha’s…
News
Mice with Rett syndrome show abnormalities in their brain circuitry that helps to control movement, which may contribute to motor dysfunction that often arises as a symptom of the disease, a new study reports. The results provide “novel in vivo [in living animals] evidence for abnormal motor circuit function”…
A set of proteins in the cerebrospinal fluid (CSF) — the fluid that flows around the brain and spinal cord — are present in unusual amounts in Rett syndrome, according to an analysis of samples from rats, mice and a small group of Rett patients. Researchers identified two particular…
From starting a Facebook fundraiser to handing out personalized information cards about Rett syndrome, supporters worldwide are marking Rett Syndrome Awareness Month, which occurs each October. Now, the International Rett Syndrome Foundation (IRSF) is offering up a host of ways people can “Go All In This October” to…
Anavex Life Sciences has been granted another U.S. patent for its lead investigational oral therapy Anavex 2-73 (blarcamesine), being developed to treat neurodevelopmental and neurological disorders, including Rett syndrome, that are driven by mutations in the MECP2 gene. This latest of a series of patents to Anavex is…
Analyzing babies’ vocalizations using machine learning could aid in the earlier diagnosis of Rett syndrome, a small study illustrates. Researchers say such vocal analysis among infants ages 6 to 11 months may help to identify signs of Rett, or fragile X syndrome, long before a child would typically…
The U.S. Food and Drug Administration (FDA) has set an action date of March 12, 2023 to decide whether or not to approve the experimental oral therapy trofinetide for Rett syndrome. If approval is granted, trofinetide would become the first treatment for Rett ever approved by the FDA.
Levels of a small RNA molecule called miR-101a may be elevated in certain brain regions of people with Rett syndrome, according to a preclinical study. The data suggest that higher miR-101a levels can alter signals sent by nerve cells, which may impair normal brain circuitry to contribute to the…
A so-called positive allosteric modulator aimed at increasing activity at a subset of muscarinic acetylcholine receptors (mAChRs) — called M1 receptors — improved behavior and eased respiratory symptoms in a mouse model of Rett syndrome, a study found. The beneficial effects of the compound, dubbed VU595, could in part…
Older age and increased muscle stiffness are linked with more severe scoliosis, a sideways curvature of the spine, in girls and women with Rett syndrome, according to a small study. Other factors associated with scoliosis severity included impaired walking and difficulties going up and down stairs. No link…
Recent Posts
- On a 15-trail accessible challenge with my daughter with Rett syndrome
- Connecting with others in the Rett community on ‘The Purple Couch’
- Lab findings support Rett gene therapy advancing in late-stage clinical trials
- What would fly for my daughter with Rett syndrome on Halloween
- ‘Rett-like’ label may offer little guidance for patients, study finds
- Taking care of a child with Rett is tiring, but not as much as having it
- Why my husband continues to fight for a cure for Rett syndrome
- ‘Alternative’ genetic cause for Rett found in case of Swedish girl
- How ‘Silent Angels’ and Julia Roberts put Rett syndrome on the map, part 2
- Targeting brain receptor yields little benefit in Rett syndrome mice