Key points Sickle cell disease (SCD) occurs when a DNA base pair substitution in the β-globin gene results in the formation of hemoglobin S, which polymerizes when deoxygenated, leading to deformation ...
Dr. Donald Kohn has been developing gene therapies for rare pediatric immune disorders for over 30 years. This week, his role in a clinical trial has culminated in the first-ever U.S. Food and Drug ...
With an end-of-week green light from the FDA, Rocket Pharmaceuticals has officially broken into commercial orbit. Still, the company is taking a more measured approach for the launch of its new gene ...
The rare pediatric disease affects approximately one in one million children globally. Mutations in the ITGB2 gene disrupt ...
Key takeawaysSevere leukocyte adhesion deficiency-I is a rare genetic disease that prevents white blood cells from functioning normally, leaving ...
Christos Soteriou was 29 when he needed a quadruple bypass surgery.
It involves substances “not for human use.” ...
In autumn 2019, the mood within the field of dementia research was bleak. Multiple late-stage clinical trials had ended in ...
One ALS patient received TRE-515 under the FDA program last year, and early signs suggest the therapy may help stabilize ...
Sarepta Therapeutics has come under pressure following the deaths of three patients using its treatments.
Results from pediatric patients in the SAVE Registry shows no device-related adverse events or immunosuppression and continued strong survival data ...
A quiet signal inside a tumor may soon guide how surgeons treat early lung cancer. Researchers now report that hidden genetic ...
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