Diseases, Conditions, Syndromes

New gene therapy poised to transform care for spinal muscular atrophy

The University of Rochester Medical Center (URMC) has been tapped as one of the first institutions in the U.S. to offer a new gene replacement therapy to treat spinal muscular atrophy (SMA). The new treatment can be delivered ...

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.

Medical research

Spinal organoids mimic neurodegenerative disease

'Organoids' that mimic the developing spinal cord could assist research and drug development for neurodegenerative diseases such as spinal muscular atrophy and amyotrophic lateral sclerosis.

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