Did you know that sickle cell disease occurs in one out of 150 Jamaican births? This is a very high risk of having SCD in comparison to African-Americans, where one…
Are prime-time TV medical-dramas truly distorting patient expectations? We don't think so. Do you? Medical dramas aren't the only ones in today's television circuit that are incorporating rare diseases into plot lines…
The month of September is recognized as National Sickle Cell Awareness Month in the US. This is a time to spread awareness about sickle cell disease among the general public…
Happy Friday Patient Worthians! What do you know about sickle cell anemia? We have seven facts for you! Also, we have some hope-worthy nonalcoholic steatohepatitis (NASH) news to report. What does…
Biopharma.com reported sharp contrasts, with a landmark approval for a rare disease therapy alongside a high-profile trial miss, and a series of corporate moves reflecting market access and financing realities.…
The U.S. Medicaid program has taken a pioneering step to ensure that new gene therapies for sickle cell disease (SCD) are accessible to the millions of Americans who rely on…
An article over at Genetic Engineering & Biotechnology News reports that Pfizer has withdrawn its sickle cell disease medication Oxbryta after the European Medicines Agency (EMA) disclosed the deaths of…
The overwhelming majority of rare diseases and conditions have a genetic basis. This means that the disease is the result of a genetic abnormality such as a mutation. These abnormalities…
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