Zygel, an experimental cannabidiol gel that Zynerba Pharmaceuticals is developing to help normalize behaviors in people with fragile X syndrome, continues to be well tolerated up to a median treatment length of 21 months, according to new data from the open-label extension of the Phase 2/3 CONNECT-FX trial.
Note: This story was updated Aug. 12, 2022, to reflect that blarcamesine is an experimental activator. Treatment with Anavex2-73 (blarcamesine) safely and significantly eased behavioral symptoms of fragile X syndrome (FXS), and normalized some aspects of brain chemistry, in a preclinical study in mice. Anavex Life Sciences, the…
Synaptogenix and Nemours A.I. DuPont Hospital for Children have signed an agreement to collaborate on a clinical trial to test the investigational medication bryostatin-1 in people with fragile X syndrome (FXS). “We are excited to participate in a potentially breakthrough therapeutic drug strategy to address this huge…
A $90,000 grant from the FRAXA Research Foundation will fund a preclinical study that aims to assess the potential benefits of a diet that is high in fat and low in carbohydrates for fragile X syndrome using mice and cell models. A high fat/low carbohydrate diet is known to be…
Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…
As early as 12 months of age, children with fragile X syndrome (FXS) display signs of social anxiety, a study indicates. The study, “Early behavioral and physiological markers of social anxiety in infants with fragile X syndrome,” was published in the Journal of Neurodevelopmental Disorders. Anxiety disorders…
The anticonvulsant carbamazepine can normalize behavior and memory in a mouse model of fragile X syndrome (FXS), suggesting that this medication might be re-purposed to treat people with this disorder, according to a study. The study, “Carbamazepine Restores Neuronal Signaling, Protein Synthesis, and Cognitive Function…
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases. “The RareLaunch program is central to NORD’s mission and history — community…
The investigational treatment BPN14770 improved language skills, cognition, and caregiver-assessed daily functioning ability in adults with fragile X syndrome, top-line results of a Phase 2 clinical trial show. “We are very excited about the results of this study,” Mark Gurney PhD, said in a press release. Gurney is…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…
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