Two of Opus's seven gene therapy assets are in clinical trials, with additional programs expected to enter the clinic.
A Northwestern Medicine study has uncovered a promising new therapeutic approach for a rare genetic brain disorder, according ...
A long-established drug with a diverse medical history is drawing new attention for its potential role in treating an ...
Experiments on an ultra-rare genetic mutation that causes neurodegeneration in children have helped uncover a new mechanism by which brain cells die. A team led by scientists at the German research ...
A newly identified and rare genetic variant slows the growth of mutated blood stem cells and reduces the risk of leukemia.
Researchers have identified specific genes containing rare mutations that significantly increase the likelihood of developing attention deficit hyperactivity disorder. These findings, published in the ...
A new study by UCLA scientists adds to the understanding of the genetic architecture of schizophrenia. Past research has shown the impact of commonly occurring genetic variants on a person's risk of ...
Tiffany Fransen, diagnosed with Friedreich's ataxia, faces challenges from this rare disease. The genetic disorder affects ...
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Top Medical Researchers Links Adolf Hitler to Rare Medical Disorder, Historians React
A new genetic study suggests Adolf Hitler most likely had Kallmann syndrome, a rare hormonal disorder that disrupts normal ...
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