An estimated 6,000-10,000 people within the United States are living with Rett syndrome. Prior to this month, these individuals have not had access to targeted treatment options. But this…
Prior studies have suggested that metformin, often used as a first-line treatment for people with type 2 diabetes (T2D0, could confer benefits for Rett syndrome. In particular, prior studies found…
In the United States, Orphan Drug designation is granted by the FDA to drugs focused on the treatment, prevention, or diagnosis of rare conditions; these are those affecting fewer than…
According to a story from Rett Syndrome News, the pharmaceutical company Acadia Pharmaceuticals is seeking U.S. Food and Drug Administration (FDA) approval for its investigational therapy trofinetide. It has been…
According to a story from news-medical.net, the International Rett Syndrome Foundation (IRSF) has announced the launch of its Clinical Trial Committee. The committee was implemented with the goal of helping…
The joy of watching your child grow and meet developmental milestones is not always granted to some parents. Clare’s mother, Allison, described her emotions to CHOP after noticing that Clare…
A new treatment for Rett syndrome is officially being studied in humans in Canada, meaning Canadian patients may have a new, viable treatment option in the future. Called TSHA-102, this…
In a news release from May 3rd, 2022, rare disease and cancer therapeutic development company DepYmed Inc. shared that its lead PTP1B inhibitor, designed to treat Rett syndrome, was granted…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
An article recently published in Medical Xpress outlined research into memory recall in Rett syndrome patients. Not only did it look into the underlying causes of diminished memory recall, but…
Stoke Therapeutics and Acadia Pharmaceuticals made a joint announcement this week through Business Wire of their collaboration in the development of RNA-based medicines to potentially treat rare neurodevelopmental disorders of…
Positive results were announced today by Anavex Life Sciences, which is based in New York, for its Phase 3 clinical trial of Anavex 2-73. Participants in the trial were female…
Colorado is a beautiful state for fly fishing, and it attracts many people who love the sport. A group of anglers from this state has joined together, united by both…
The Munson family is fundraising to make changes to their home, as these adaptations will make life easier for their two children Oscar and his older sister Mary. This is…
Many little girls dream of being princesses, and Michele Kingsland is no different. Well, she is a little different, because her wish was actually granted. The Make-A-Wish Foundation crowned Michele…
In a recent news release, gene therapy company Taysha Gene Therapies ("Taysha") shared the publication of preclinical data on the company's drug candidate, TSHA-102. Currently, Taysha is exploring TSHA-102 as…
According to a story from Benzinga, there are at least eight drug companies that are currently working on a treatment for Rett syndrome, a rare genetic disorder. With no cure…
During clinical trials, Independent Data Safety Monitoring Boards (DSMBs) sometimes assess trial safety, trial integrity, and how well the trials are upholding patient needs. According to Business Insider, the DSMB…
Rett syndrome is a rare neurological disorder which often presents within the first 6 months to 1.5 years of a child's life. As the condition progresses, children often lose…
As the medical field expands, so do the options for new and intriguing medical research. According to Drug Target Review, researchers from the University of California (UC) San Diego…
Did you know that zebrafish, a tropical freshwater fish, are being more commonly used to model and study human diseases? Now, says Phys.org, researchers from the Garvan Institute of Medical…
Many rare diseases are in dire need of need of viable treatments. To fix this problem, researchers have began creating and indicating existing treatments for rare diseases. An example would…
Recently, gene therapy company Taysha Gene Therapies ("Taysha") announced that its gene therapy candidate, TSHA-102, received both Orphan Drug and Rare Pediatric Disease designations from the FDA. TSHA-102, delivered via…
October is Rett Syndrome Awareness Month, and even though the pandemic has changed the way that we celebrate it, there are plenty of ways to get involved! Rettsyndrome.org has organized…
According to an article in the publication Labroots, autism spectrum disorder (ASD) may be the result of interaction between environmental and genetic factors. For instance, researchers point out that ASD…
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