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.

Genetics

Genetic defects without consequences

Many diseases are caused by genetic defects. However, their severity can vary among individual patients, so that even mild forms can occur. Scientists at the Max Planck Institute for Heart and Lung Research in Bad Nauheim ...

Genetics

Researchers discover a new form of hereditary osteoporosis

A research group headed by Professor Outi Mäkitie, University of Helsinki, identified in families with childhood-onset osteoporosis disease-causing mutations in a gene that had previously not been connected with the skeletal ...

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