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…
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