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).…
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…
The 16th NFXF International Fragile X Conference This event will cover all fragile X disorders and is the largest fragile X event in the world. Caregivers, families, patients, and medical…
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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