Twenty-six years ago today, on Sept. 17, a teenager who had received an experimental gene therapy died. His death led to needed changes in the clinical trial process while also spurring skepticism ...
As we continue our series exploring genetic medicine, both in this story and in Destiny's Child No Longer: Rewriting Genetic Fate, gene therapy stands at a crossroads. On June 16, Sarepta Therapeutics ...
CRF acquired Spark Therapeutics, Inc.’s SPK-7001 CHM gene therapy assets and is analyzing long-term data to identify ...
After 35 years of consistent pain, Allan Byamukama became one of the few individuals in the world cured of sickle cell ...
A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the burden of lifelong transfusions. A single one-time gene therapy could free ...
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