Recent research indicates that microRNAs may be a potential biomarker to diagnose and judge a Parkinson's patient's prognosis. A study titled “Deep sequencing of sncRNAs reveals hallmarks and regulatory modules…
According to a story from the National Hemophilia Foundation, the drug company uniQure has provided an update in regards to a case of hepatocellular carcinoma (HCC) that emerged in a…
In a recent press release, clinical-stage gene therapy company Rocket Pharmaceuticals, Inc. ("Rocket") shared that its investigational gene therapy candidate, RP-L201, received Priority Medicines (PRIME) designation from the European…
Just as our emotions control our the body, determining our posture and expression and tensions; our bodies are our feeling devices, with the sensory input and muscle tension in turn…
In a recent contribution to MSN, Dr. Christopher Austin, director of the NIH Advancing Translational Sciences, compared the medical community’s record-breaking approach to the COVID-19 pandemic with current efforts…
Macey Brietenback, a resident of Bel Air, Maryland, is nineteen years old and has endured various operating procedures over 70 times. WJZ has been following Macey’s struggle with her…
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…
Fatty liver disease, which can be caused by alcohol consumption or can be unrelated (nonalcoholic fatty liver disease/NAFLD), belongs to a larger group of conditions that have been labeled as…
One year ago, Dr. Audrey Odom John Chief of the Division of Infectious Diseases at Philadephia’s Children’s Hospital, treated a teenage patient for what seemed to be a new disease.…
Many rare disease patients face difficulty when it comes to diagnosis. In fact, it takes an average of five to seven years for them to finally receive the correct diagnosis.…
When I sat down with 38-year-old Adam Kemble for our interview, there was one point that he really drove home: “You have the opportunity to choose what you let define…
When Nikki was growing up, she was plagued by frequent nosebleeds, and they weren't minor ones at that. They were serious and frequent enough to cause significant disruptions to her…
The latest gene editing technology, prime editing, expands the 'genetic toolbox' for more precisely creating disease models and correcting genetic problems, scientists say. Read the source article at sciencedaily.com
Click here for updates on this story BEAVERTON, Oregon ( KPTV ) — A 5-year-old Beaverton boy is fighting a rare disorder called Krabbe disease. His parents say it’s possible…
According to a story from businesswire.com, the pharmaceutical company Bayer has recently announced the release of results from its phase IV trial. This trial evaluated the effects of the drug…
In a press release from early March 2021, radiopharmaceutical therapy company Molecular Targeting Technologies, Inc. ("MTTI") shared that the FDA approved its Investigational New Drug (IND) application for EBTATE.…
Caroline is the adorable three-year-old daughter of Kevin and Kelly Brennan Culver, and she was diagnosed with juvenile dermatomyositis (JDM) about a year ago. This means that she requires an…
Leigh syndrome is a rare mitochondrial disease that is not yet well understood by medical professionals. When this lack of comprehension exists, it is impossible to create treatments or improve…
Lisa Stockman Mauriello of Summit, New Jersey, is not alone in her struggle to gain access to the company’s experimental ALS drug, tofersen. In fact, according to a recent article…
According to a story from PR Newswire, the biotechnology company Ionis Pharmaceuticals recently announced promising findings from its phase 2 clinical trial. This clinical trial was evaluating the company's experimental…
Rare disease patients face a number of obstacles when it comes to getting the proper diagnosis and treatment. There's a lot of work being done to address these problems, some…
NORD (the National Organization for Rare Disorders), through PR Newswire, recently announced the results of a new study that reveals the total number of generics, orphan products, and biosimilars currently…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
As reported in Business Wire; VerSIM Life, a company that uses computational sciences to create novel machine learning to aid drug development, has announced that they are launching a pharmaceutical…
Did you know that there are a variety of conditions which can occur in tandem, or even because of, another condition? According to Renal & Urology News, this may be…
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