Often misdiagnosed as other conditions such as Rett syndrome or cerebral palsy, DDX3X mutations cause both cognitive and physical difficulties. Since only an estimated 700 patients have been diagnosed…
In the past, arimoclomol has not always shown treatment promise. For example, the drug failed to meet primary or secondary endpoints in a clinical trial evaluating arimoclomol for patients with…
According to a story from GlobeNewswire, the gene therapy company LEXEO Therapeutics has recently announced that its investigational gene therapy LX2006 has been granted both Orphan Drug designation and Rare…
Unfortunately, many molecules which drive cancer development and growth are considered "undruggable." In short, this means that drugs are unable to bind to or address these molecules due to size,…
Even though my boyfriend's mom works at an eye doctor, I'm still kind of afraid to get eye exams - and I'm not alone. Unfortunately, most people do not receive frequent enough…
July is recognized as Juvenile Idiopathic Arthritis Awareness Month. Though arthritis is often considered an illness that is associated with old age, it is estimated that around 300,000 children in…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
The European Hematology Association (EHA) held its 2021 Virtual Congress from June 9-17, 2021, though the insights will be available until mid-August 2021. During the Congress, researchers discussed new…
A new study, funded by the NIH and conducted by Vanderbilt University Medical Center (VUMC), is working to assess allergic reactions to COVID-19 vaccines. The trial will include individuals between…
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