Genetics

Scientists discover link between ALS genes

The enzyme Gemin3 was identified as the molecular 'bridge' between genes whose mutation or disruption causes amyotrophic lateral sclerosis (ALS), according to a new study in Nature's Scientific Reports from scientists at ...

Medications

Novartis eyes give-away of costly therapy for babies

Swiss pharmaceutical company Novartis is preparing to give away 100 doses of the world's most expensive drug, which treats a rare childhood disorder, but its recipient selection process has drawn criticism.

Diseases, Conditions, Syndromes

High-tech method for uniquely targeted gene therapy

Neuroscientists at Lund University in Sweden have developed a new technology that engineers the shell of a virus to deliver gene therapy to the exact cell type in the body that needs to be treated. The researchers believe ...

Diseases, Conditions, Syndromes

Study could aid degenerative disease therapies

Fresh insights into how nerves connect with muscles in the body could aid the development of therapies to treat neurodegenerative diseases.

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.

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