Acknowledgement: This article was shared with Patient Worthy by our friends at Stork Genetics. To see the original article, please click here. For more information about women affected by X-linked…
Spinogenix has reported that its Phase II trial of SPG601 for Fragile X Syndrome (FSX) has met its primary goal, according to Clinical Trials Arena. FXS is a genetic disorder…
In the United States, Orphan Drug designation is granted by the FDA to drugs or biologics designed to treat, diagnose, or prevent a rare condition affecting fewer than 200,000 people…
Right now, there are no available therapies for people living with Fragile X syndrome (FXS). However, clinical-stage biotechnology company Connecta Therapeutics (“Connecta”) is working to change that. In a recent…
When it comes to rare diseases, advancing research is crucial to developing a better understanding of the disease and how it can be treated. However, it can sometimes be difficult…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions. A rare condition is one affecting fewer…
Before reading, don't forget to head to Part 1 to learn more about Quris' founder Isaac Bentwich, why Quris was developed, and how the company is using its innovative bio-AI drug development…
Many people laud the accomplishments of clinical trials – and for, in some cases, good reason. Clinical trials have been crucial in identifying and developing therapeutic options for patients to…
Lysogene has just announced a new study has been published in the journal EMBO Molecular Medicine which demonstrates that DGKk is able to produce long-term rescue of fragile X syndrome (FXS).…
Cannabidiol (CBD) has recently been lauded for its many uses, ranging from stress relief to seizure reductions. Specialty pharmaceuticals company Zynerba Pharmaceuticals (“Zynerba”) developed an experimental cannabidiol gel called Zygel…
Healx has just announced that they've been given Investigational New Drug FDA approval for HLX-0201 for Fragile X syndrome. This means they are able to move forward with their Phase 2a…
As many patients within the rare disease community know, the diagnostic odyssey can be long and arduous. But what if burgeoning technologies, like artificial intelligence (AI) and its branches (machine…
The Orange County Guide features doctors’ articles in each issue. Dr. Justin West’s search to cure his son’s rare disease will be familiar to families also in search of…
According to Fragile X News Today, a new registry recently launched within the United States to spur research into gene premutations linked to fragile X syndrome (FXS). Currently, the…
People with genetic diseases or disorders may experience differences on a physiological level. For example, shares Medical XPress, fragile X syndrome (FXS) results in less primary cilia. Since primary cilia…
According to a story from BioSpace, the drug company Zynerba Pharmaceuticals Inc. recently released the results from a clinical trial. This trial was testing the company's product Zygel™ (CBD gel)…
By Danielle Bradshaw from In The Cloud Copy Expressive language sampling, or ELS, could potentially help researchers better measure how well treatments work for patients with fragile X syndrome or…
According to MIT News, scientists recently discovered that inhibiting levels of the GSK3 alpha enzyme could reduce, or even reverse, symptoms of fragile X syndrome. Their study, which utilized mouse…
Christopher Cowan and his lab at the Medical University of South Carolina recently conducted a study in mice in order to understand how defective MEF2C genes impact brain development. A…
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…
As originally reported in the Cambridge Independent; rare disease treatment is full of potential. In part, because there aren’t many. This is not for lack of ability, but for lack…
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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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