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

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 ...

Medical research

Study gives clues to causes of motor neurone disease

(Medical Xpress)—Scientists at the University of Bath are one step further to understanding the role of one of the proteins that causes the neurodegenerative disorder, Amyotrophic Lateral Sclerosis (ALS), also known as ...

Medical research

Researchers review muscular dystrophy therapies

Leading muscular dystrophy researcher Dean Burkin, of the University of Nevada School of Medicine summarizes the impact of a new protein therapeutic, MG53, for the treatment of Duchenne muscular dystrophy in an article published ...

Neuroscience

Newborn screening for DMD shows promise as an international model

Investigators at Nationwide Children's Hospital, working with the DNA Sequencing Core Facility at the University of Utah, have developed an approach to newborn screening (NBS) for the life-threatening genetic disorder, Duchenne ...

Diseases, Conditions, Syndromes

Some muscular dystrophy patients at increased risk for cancer

People who have the most common type of adult muscular dystrophy also have a higher risk of getting cancer, according to a paper published today in the Journal of the American Medical Association.

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