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 ...

Medical research

Tongue-on-a-chip provides insight into genetic diseases

Muscular dystrophy is a group of rare genetic diseases that cause progressive muscle weakness and deterioration. For decades, researchers have struggled to understand how the disease works and to find suitable treatments.

Medical research

Duchenne muscular dystrophy is a stem cell disease

A new study from The Ottawa Hospital and the University of Ottawa is poised to completely change our understanding of Duchenne muscular dystrophy and pave the way for far more effective treatments.

Medical research

Mutant protein in muscle linked to neuromuscular disorder

Sometimes known as Kennedy's disease, spinal and bulbar muscular atrophy (SBMA) is a rare inherited neuromuscular disorder characterized by slowly progressive muscle weakness and atrophy. Researchers have long considered ...

Neuroscience

Team finds melatonin delays ALS symptom onset and death in mice

Melatonin injections delayed symptom onset and reduced mortality in a mouse model of the neurodegenerative condition amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease, according to a new study by researchers at ...

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