Medications

FDA approves $2M medicine, most expensive ever

U.S. regulators have approved the most expensive medicine ever, for a rare disorder that destroys a baby's muscle control and kills nearly all of those with the most common type of the disease within a couple of years.

Neuroscience

ALS research reveals new treatment approach

On June 2, 1941, at the age of 37, baseball player Lou Gehrig succumbed to amyotrophic lateral sclerosis (ALS), a progressive muscle degeneration disease. Today, nearly 80 years following his death, scientists are still searching ...

Medical research

Biotech startup uses nanoparticles to induce immune tolerance

More than 100 approved drugs in the U.S. warn of immune-related side effects on their labels. Countless others never make it onto shelves because of unwanted immune responses that can harm patients and limit the effectiveness ...

Genetics

Researchers discover new disease

A new immunodeficiency disease caused by a novel genetic mutation has been identified by researchers at the University of Colorado Anschutz Medical Campus providing unique insights into cell biology.

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Gene therapy

Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease, such as a hereditary disease in which a deleterious mutant allele is replaced with a functional one. Although the technology is still in its infancy, it has been used with some success. Antisense therapy is not strictly a form of gene therapy, but is a genetically-mediated therapy and is often considered together with other methods.

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