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…
Rett syndrome is a rare neurological disorder which often presents within the first 6 months to 1.5 years of a child's life. As the condition progresses, children often lose…
Tanner McLeod has been searching for a stem cell donor for a long time. He was diagnosed with sideroblastic anemia at just six months old and has required a transplant…
Recent results of a Turkish trial have demonstrated the need for further research into IFN-gamma as a treatment for Friedreich's ataxia (FA). This study showed that the therapy resulted in…
While gene therapy is a burgeoning technique to treat a number of rare genetic conditions, there are still a few complexities associated with this treatment option. How safe and effective…
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