The Ontario Rett Syndrome Association (ORSA) announced the awarding of two grants to support research into Rett syndrome in Canada. According to an ORSA press release, grants totaling CA$78,750 (about $62,800) will be given to two selected projects. The funding comes from The Hope Fund, which ORSA…
News
A music-based intervention helped to normalize certain social behaviors in a mouse model of Rett syndrome, researchers reported. Findings also suggested that repeated, regular exposure to music altered the activity of certain genes in particular brain regions, yielding clues as to the biologic mechanisms through which the intervention works. The…
A first-generation university student has been awarded a six-year, $466,669 grant from the National Institutes of Health (NIH) to fund his doctoral and postdoctoral research into brain-derived neurotrophic factor (BDNF), a protein that plays a role in Rett syndrome. The Virginia Tech student, Raymundo Hernandez, called the NIH grant…
Shape Therapeutics has raised $112 million in financing to support work into developing its portfolio of RNA technologies to potentially treat genetic diseases, including Rett syndrome. “We have witnessed the power of [messenger RNA] in the vaccine space and are now entering a new era where we can…
The molecular mechanism by which the protein MeCP2 recognizes and binds to CA repeats — repetitive short sequences of DNA that are found throughout the genome — has been unraveled in a new study that sheds light on the causes of Rett syndrome. Researchers in France discovered that MeCP2…
Treatment with a protein called CNF1 produced different results in two different mouse models of Rett syndrome, demonstrating a need to test therapeutic approaches across disease models, according to a study. Specifically, the CNF1 protein was able to rescue cognitive deficits, but not motor difficulties, in a mouse model…
Using a virtual avatar to guide users during attention and memory tasks significantly improves the performance of people with Rett syndrome, according to a small study from Italy. The human-like interactive agent likely acts as a social motivator for these  patients, who often focus on the face of parents and…
The lack of a working MeCP2 protein — the most common cause of Rett syndrome — skews the immune system toward a proinflammatory state in the central nervous system (CNS, brain and spinal cord), a study in mice suggests. According to its scientists, this finding helps in understanding the…
At its first virtual investor event, biotech company Centogene set a bold mission: to cure 100 rare diseases within the next decade. A leader in the field of genetic diagnostics, Centogene used the June 22 event to present its strategic priorities, outlining its plans to speed the discovery…
The use of microRNA molecules that targeted a protein receptor important for nerve cell growth led to improved behaviors and motor function in a Rett syndrome mouse model, a study shows. These molecules were carried by tiny vesicles called exosomes, which were derived from human urinary stem cells. The…
Recent Posts
- Fenfluramine reduces hard-to-treat seizures in 3 girls with Rett syndrome
- ‘Spread the Word Day’ reminds me of our own encounter with the R-word
- New study supports switching between Daybue liquid and powder forms
- Leaky blood vessels in the brain linked to Rett syndrome: Study
- A song, an obit, and a special needs parent meltdown
- The importance of raising awareness of disabilities at school
- Boosting production of mutant protein causing Rett may be new treatment
- What we’ve learned in 29 years as Rett syndrome parents
- Acadia to challenge negative opinion on Daybue approval in EU
- I’ve been on the lookout for the look that says, ‘Enough is enough’