Neuroscience

Target identified for rare inherited neurological disease in men

Researchers at University of California, San Diego School of Medicine have identified the mechanism by which a rare, inherited neurodegenerative disease causes often crippling muscle weakness in men, in addition to reduced ...

Medical research

Are my muscular dystrophy drugs working?

People with muscular dystrophy could one day assess the effectiveness of their medication with the help of a smartphone-linked device, a new study in mice suggests. The study used a new method to process ultrasound imaging ...

Medical research

RNA editing technique treats severe form of muscular dystrophy

An RNA editing technique called "exon skipping" has shown preliminary success in treating a rare and severe form of muscular dystrophy that currently has no treatment, based on a new study from Northwestern Medicine and the ...

Medications

FDA OKs first treatment for rare genetic disorder

The Food and Drug Administration on Friday approved the first treatment for children and adults with spinal muscular atrophy, a rare genetic disorder marked by progressive muscle weakness that's the most common genetic cause ...

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