Diseases, Conditions, Syndromes

Toward a reliable oral treatment for sickle cell disease

For the millions of people worldwide who have sickle cell disease, there are only a few treatment options, which include risky bone marrow transplants, gene therapy or other treatments that address a subset of symptoms. Today, ...

Genetics

Defining precision medicine

When President Obama uttered the words "Precision Medicine" in the state-of-the-union address, I scoffed at a politician's finally noticing a field that's been around for decades: medical genetics. Was it another case of ...

Diseases, Conditions, Syndromes

A better treatment for sickle cell disease

Sickle cell disease is the most prevalent inherited blood disorder in the world, affecting 70,000 to 100,000 Americans. However, it is considered an orphan disease, meaning it impacts less than 200,000 people nationally, ...

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