According to a story from Wapakoneta Daily News, the drug companies ACADIA Pharmaceuticals Inc. and Neuren Pharmaceuticals Limited have recently announced that its investigational product candidate trofinetide has earned Rare…
Zynerba Pharmaceuticals has recently announced that they have finished the patient screening for their clinical trial of Zygel, a treatment for Fragile X syndrome. Zygel is a CBD gel that…
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Stéphanie Tomé is an investigator at the Sorbonne Université in Paris, France. A recent article in PacBio describes Tomé’s research into a disease that becomes progressively worse with each generation.…
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A recent announcement by Atomico, one of Europe’s largest venture capital firms based in London, introduced its new partner Healx. The startup can boast of having the world’s most comprehensive…
Results of a study reported in PLOS ONE that was conducted by a team of scientists indicate that the experimental drug mavoglurant improves responsiveness and eye gaze for patients with…
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A guest speaker at the 2019 Rare Disease Forum recently held at the North Carolina Biotechnology Center acknowledged that screening is necessary to justify the need for a particular…
According to a story from Venture Beat, the company Healx has succesfully raised $56 million in funds that will go towards using artificial intelligence (AI) to identify new potential treatments…
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Alzheimer’s disease (AD) is a neurodegenerative disease and the major cause of impaired memory affecting the older population. Reported cases are rapidly increasing. So far, clinical trials have failed…
According to a story from Medical Xpress, a recent study has revealed that a certain genetic premutation affected the FMR1 gene could be a risk factor for far more health…
According to a story from globenewswire.com, the drug company Zynerba Pharmaceuticals, Inc. has announced that the data from its phase 2 open label trial has been officially published. This clinical…
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Researchers at the University of Tokyo have discovered that one segment of DNA is responsible for four rare diseases. As reported in Science Daily, the university team also believes that…
According to a story from globenewswire.com, the drug developer Zynerba Pharmaceuticals recently announced that the company has been awarded Fast Track Designation from the US Food and Drug Administration (FDA).…
According to the publication from the London Free Press, a new technique in development at Lawson Health Research Institute uses epigenetics to more closely study genetic expression in humans. Epigenetics…
Cardiff University in Wales has just announced the launch of their new Medicines Discovery Institute. It’s goal? To help accelerate the development of novel therapies for those living with conditions…
As discussed in a recent article on PatientWorthy the FDNA has recently developed a new kind of algorithm which can diagnose rare genetic conditions such as Angelman syndrome, Williams syndrome, and Fragile X syndrome using…
According to a story from pm360online.com, the drug developer Zynerba Pharmaceuticals, Inc., recently announced the release of data from an open label, 12 month study of the company's experimental product…
Angelman syndrome impedes the brain's ability to distinguish background noise from whats important. This is a result of a reduced tonic inhibition, caused by a loss of function of the…
Researchers at the University of Pennsylvania have studied the genetics of a class of neurological disorders that includes Huntington’s disease and Fragile X Syndrome. They found that there were similarities…
Having a child was an exciting moment for the Blackwood family. Their son, Sam, is now nine years old. It was around his first birthday, however, that they started to…
Zynerba Pharmaceuticals, Inc. has announced their plans to begin a clinical trial of Cannabidiol in children and teenagers who have Fragile X syndrome. The study, called CONNECT-FX, is expected to…
According to a story from Medical Xpress, a recent study is revealing pronounced differences in the brains of people with fragile x syndrome when compared to the brains of people…
According to a story from pharmiweb.com, the pharmaceutical company Ovid Therapeutics announced recently that the U.S. Food and Drug Administration (FDA) has given its drug candidate OV101 Fast Track designation…
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